ORPHELIA Pharma and University of Birmingham strengthen their collaboration to support the registration of KIZFIZO®

May 22nd, 2024.  The University of Birmingham and Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of orphan paediatric medicines, are pleased to announce the extension of their collaboration to leverage patient-level data of the BEACON refractory or relapsed neuroblastoma trial to support the marketing authorisation of KIZFIZO.

Neuroblastoma is a rare type of cancer usually diagnosed in children under 2 years of age, and for which age-specific therapeutic options are needed. 

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is the first ready-to-use oral liquid paediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carries a very poor prognosis.

The BEACON trial (NCT02308527) was a hypothesis-generating phase II trial that served to identify active regimens in relapsed or refractory neuroblastoma, some of which are now being further investigated. The trial recruited paediatric patients between 2013 and 2021 and involved the University of Birmingham as the sponsor, the European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

KIZFIZO was not available in time for the BEACON trial but Orphelia Pharma and the University of Birmingham are collaborating to assess the clinical benefit and the safety of temozolomide-based regimens in relapsed or refractory high-risk neuroblastoma patients. Overall, anonymised data from the BEACON trial are being used to support the registration dossier of KIZFIZO, according to a data sharing agreement drawn up by University of Birmingham Enterprise. 

BEACON-2 is the follow-on trial that will test bevacizumab and dinutuximab beta in combination with the backbone chemotherapy temozolomide and irinotecan. As part of its ongoing commitment to child cancer, Orphelia Pharma has also agreed to support the new BEACON-2 trial, also sponsored by University of Birmingham, by making KIZFIZO available as a child-friendly temozolomide option, in addition to the standard form of temozolomide, where participating investigators can have access to this medicine upon regulatory approval of its use as part of the BEACON-2 trial.

Jeremy Bastid, Chief Development Officer at Orphelia Pharma, commented: “The support of the University of Birmingham has been invaluable to us. The BEACON data has already demonstrated a clear clinical benefit and an improved overall survival over historical controls.”

Dr Lucas Moreno, Head of Paediatric Haematology and Oncology at Vall d’Hebron University Hospital, Barcelona, Spain and Chief Investigator for BEACON said: “We are delighted that the data generated could not only delineate the best treatment options for those patients, but also support the registration of a new paediatric formulation of temozolomide, which is much sought-after for our younger patients.”

Professor Amos Burke, Director of the Cancer Research Clinical Trials Unit concluded: “A liquid formulation of temozolomide is welcomed for younger patients and the use of data from the standard capsule formulation of temozolomide used in BEACON to support registration for a new liquid formulation demonstrates the importance of the collaborative use of results from this practice-changing trial.”

About University of Birmingham

The University of Birmingham is ranked amongst the world’s top 100 institutions.  Its work brings people from across the world to Birmingham, including researchers, teachers and more than 6,500 international students from over 150 countries. 

University of Birmingham Enterprise helps students and researchers turn their ideas into new services, products and enterprises that meet real-world needs. We also provide incubation and bio-incubation, support innovators and entrepreneurs with mentoring, advice and training and manage the University’s Academic Consultancy Service.  Follow us on LinkedIn and X.

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of paediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the paediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.

For further information: www.orphelia-pharma.eu, LinkedIn

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid paediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received an Orphan Drug Designation from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

About the BEACON trial

Results from the BEACON trial were published in the Journal of Clinical Oncology and found that adding bevacizumab alongside chemotherapy treatments including temozolomide had an increase in the likelihood of responding to treatment, from 18% among those who only had the established therapy to 26% for those with the additional drug. Patients who received Bevacizumab additionally had better one year progression-free survival rates.

Orphelia Pharma secures European patent covering KIGABEQ®, the first pediatric formulation of vigabatrin

Company benefits from protection period until 2036

Paris and Lyon, France, April 18th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, today announces that the European Patent Office (EPO) has issued an intention to grant patent number EP3426230 for KIGABEQ®.

This European patent and its granted US counterpart (US 10,813,898), entitled “Solid dosage forms of vigabatrin”, are solely owned by Orphelia Pharma. Based on the filing date of the priority PCT application, the patent protection is expected to last until 2036.

KIGABEQ® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug. Available as 100 mg and 500 mg scored soluble tablets for oral and (naso)gastric administration, KIGABEQ® is indicated in the first-line treatment of infantile spasms, an extremely severe early childhood encephalopathic epilepsy. KIGABEQ® has been developed exclusively for children aged between 1 month and 6 years and has been granted a Paediatric Use Marketing Authorization (PUMA) in Europe.

“Following the issuance of the US patent, the grant of the EU patent highlights the innovative nature of KIGABEQ® formulation” said Hugues Bienaymé, General Manager of Orphelia Pharma. “This patent underscores our dedication to innovation which lies at the heart of our development strategy towards better pediatric medicines.”

About KIGABEQ®

KIGABEQ® is the first pediatric formulation of vigabatrin, an essential anti-epileptic drug. Presented as soluble and scored tablets of 100 mg and 500 mg for oral or (naso)gastric administration, KIGABEQ® is notably indicated for the treatment of infantile spasms (West syndrome), an extremely serious epileptic encephalopathy of the infant. KIGABEQ® was developed exclusively for children and has been granted European Pediatric Marketing Authorization (PUMA).

For further information: https://www.ema.europa.eu/en/medicines/human/EPAR/kigabeq

About infantile spasms

West syndrome, or infantile spasms, is an extremely serious epileptic encephalopathy in infants that combines epileptic spasms, psychomotor deterioration and a hypsarrhythmic type electroencephalogram. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in an infant with previously normal development or with pre-existing mental retardation; in all cases, infantile spasms severely hamper the psychomotor development. Pharmacological treatment should be started quickly to allow the spasms to stop and improve the prognosis.

For further information: https://www.orpha.net/fr/disease/detail/3451

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.

For further information: www.orphelia-pharma.eu, LinkedIn

International Drug Repurposing Conference 2024, 6-7th March 2024, Barcelona

The International Drug Repurposing Conference (#iDR24): Bridging Boundaries for Innovative Drug Repurposing, co-organized by REMEDi4ALL Consortium, Beacon for Rare Diseases, and MeRIT will be held on 6-7 March 2024 in Barcelona. 

Drug repurposing consists of finding new therapeutic uses for existing medicines, building on the knowledge previously generated on safety and effectiveness for a specific drug. This means that scientific knowledge and resources are used efficiently, and research can move faster to provide patients with innovative therapeutic options where no treatments are available.

The conference showcases how patient communities, researchers, industry, and health authorities can benefit from cutting-edge drug repurposing.

Laurent Martin, ORPHELIA Pharma’s Chief Pharmaceutical Affairs Officer, will contribute to the discussion with the story and market access of Kigabeq, the first pediatric formulation of vigabatrin for the treatment of a rare form of encephalopathic epilepsy: West syndrome.

More information can be found at: https://remedi4all.org/international-drug-repurposing-conference/

LinkedIn: https://www.linkedin.com/feed/update/urn:li:share:7170693183050346496/

ORPHELIA Pharma raises awareness for childhood cancer

Today, February 15th, is the International Childhood Cancer Day. One out of 440 children is affected by cancer, i.e. 2 300 young patients every year in France. Although 80% of children are cured, one out of five with certain difficult indications does not survive. This ICCD is the opportunity for ORPHELIA Pharma to re-express its commitment to advancing care, treatments and research in the field of pediatric oncology.

ORPHELIA Pharma announces the publication of two scientific articles on KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma

Paris and Lyon, January 9th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announces the publication of two scientific articles about KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma, which is under registration review under centralised procedure by the EMA.

The first article, entitled Dispensing Oral Temozolomide in Children: “Precision and Stability of a Novel and Ready to Use Liquid Formulation in Comparison with Capsule Derived Mixtures”, published in Pharmaceutics, demonstrates that the clinical practice of administering temozolomide capsules (Temodal) by mixing the content with food or drinks results in significant underexposure compared to administering the ready-to-use KIZFIZO 40 mg/ml oral suspension.

Tests for the accuracy of the administered dose and stability of temozolomide were performed with the capsule contents mixed with food vehicles (apple juice, applesauce, cream, milk and mashed potatoes) and compared to an equivalent dose of KIZFIZO. The dose delivered was systematically lower with capsule-based preparations, regardless of the vehicle used.

In addition, certain food vehicles (dessert cream, milk, mashed potatoes) had a significant effect on the chemical degradation of temozolomide, with the significant increase of its hydrolysis product, amino-imidazole-carboxamide (AIC).

The second article, also published in Pharmaceutics, as : A Multicenter Randomized Bioequivalence Study of a Novel Ready-to-Use Temozolomide Oral Suspension vs. Temozolomide Capsules”, demonstrates the bioequivalence between KIZFIZO oral suspension and the capsule form of temozolomide (Temodal).

In conclusion, the administration of KIZFIZO, which is bioequivalent with the capsule form of temozolomide, avoids the misuse associated with the administration of capsules in the paediatric population. This ready to use oral liquid formulation, which is taste masked and ensures the chemical stability of temozolomide, accurately delivers the prescribed dose.

About KIZFIZO® 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received an Orphan Drug Designation from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.

For further information: www.orphelia-pharma.eu, LinkedIn

Orphelia Pharma secures US patent covering KIZFIZO®, first drinkable formulation of temozolomide

Company benefits from protection period until 2038

Paris and Lyon, France, November 29, 2023 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, today announces that the US Patent and Trademark Office (USPTO) has issued patent US11/730,732 for KIZFIZO®, the first pediatric and ready-to-use drinkable formulation of temozolomide.

KIZFIZO (temozolomide oral suspension, 40 mg/ml), known as Ped-TMZ or KIMOZO during its clinical development and ongoing early access programs, is specifically designed for use in the treatment of children with relapsed or refractory high-risk neuroblastoma, oncology indications with a very poor prognosis. This taste-masked oral suspension was developed for children: it allows a precise dose to be administered orally or via a nasogastric tube in a small volume. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last six years.

“Following the issuance of the European patent in 2021, this grant of the US patent once again highlights the innovation in the formulation of KIZFIZO, which lies in the discovery of an essential excipient improving the stability and the rheological properties of the suspension,” said Jeremy Bastid, chief development officer at Orphelia Pharma. “This US patent complements the European one already issued and the Orphan Drug Designation (ODD) obtained in the US and Europe for temozolomide in the treatment of neuroblastoma. Therefore, we benefit from a double exclusivity protection for KIZFIZO in these territories.”

“There is a major hospital need for an oral formulation of temozolomide, but attempts to generate such liquid formulations have been unsatisfactory, with low concentrations leading to large administration volumes, short shelf-lives and erratic recrystallization,” said Maxime Annereau, pharmacist at Gustave Roussy and co-inventor of the patent. “Our work, carried out jointly with the Orphelia Pharma team, led to the identification of a formulation with a high concentration of temozolomide while controlling its polymorphic transition process.”

Jeremy Bastid added: “Temozolomide is an essential component in the treatment armamentarium of refractory or relapsed high-risk neuroblastoma, a disease that affects very young children. KIZFIZO addresses a major unmet medical need. We will now focus our efforts on making KIZFIZO, which is already accessible under the ongoing early access program, available quickly to all patients; we have recently submitted an application for marketing authorization in Europe.”

The granted US (US11/730,732) and European (EP3613436) patents ‘oral suspension of temozolomide’ are co-owned by Orphelia Pharma and Gustave Roussy. Based on the filing date of the priority application, the patent protection is expected to last until 2038. This protection complements the ODDs already obtained in these territories.
 
About KIZFIZO® 40 mg/ml
KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.
In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.
KIZFIZO has received an ODD from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.
 
About Orphelia Pharma
Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.
For further information: www.orphelia-pharma.eu, LinkedIn
 
About Gustave Roussy
Ranked as the leading European Cancer Center and third in the world, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering with cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. It is a source of diagnostic and therapeutic advances. Per year, it caters for almost 50,000 patients, including 3,500 pediatric patients, and its approach is one that integrates research, patient care and teaching. It is specialized in the treatment of rare cancers and complex tumors and it treats all cancers in patients of any age. Its care is personalized and combines the most advanced medical methods with an appreciation of the patient’s human requirements. In addition to the quality of treatment offered, the physical, psychological and social aspects of the patient’s life are respected. 4,100 professionals work on its two campuses: Villejuif and Chevilly-Larue. Gustave Roussy brings together the skills which are essential for the highest quality research in oncology: 40% of patients treated are included in clinical studies. 
For further information: www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram

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ORPHELIA Pharma to support the European paediatric Soft Tissue Sarcoma Study Group winter meeting.

The European paediatric Soft Tissue Sarcoma Study Group (EpSSG; https://www.epssgassociation.it/) is holding its winter meeting on November 29th – December 1st, 2023, in Barcelona, Spain.

Being committed to the development of essential medicines for rare pediatric cancers, ORPHELIA Pharma is proud to support this event as a sponsor.

EpSSG Association exists to promote and manage clinical trials, encourage and facilitate clinical and basic science research, foster optimal standards of care, organize educational meetings for its members and other professionals, and advocate for patients with soft tissue sarcoma (STS). STS account for approximately 8% of all pediatric cancers. They are divided into two main groups :  rhabdomyosarcoma (approximately 60% of STS in children) and non-rhabdomyosarcoma soft tissue sarcomas (this includes a large number of different tumor types which together account for about 40% of STS). Additional information about this event can be found here : https://www.epssgassociation.it/activities/epssg-meetings/upcoming-meeting/winter-meeting/,229

Medicines for Europe : 5th Value Added Medicines Conference, 7th November 2023, Brussels

The Medicines for Europe association represent the pharmaceutical companies supplying the largest share of medicines across Europe and is the voice of the generic, biosimilar and value-added industries.

It holds its 5th conference on value-added medicines on November 7th, 2023, in Brussels. One of the topics to be addressed in this conference is repurposing of medicines, which has emerged as an important strategy to address unmet medical need, especially in the pediatric field.

Laurent Martin, Orphelia Pharma’s Chief Pharmaceutical Affairs Officer, will contribute to the discussion with the story and market access of Kigabeq, the first pediatric formulation of vigabatrin for the treatment of a rare form of encephalopathic epilepsy: West syndrome.

An event not to be missed! More information can be found at: https://www.medicinesforeurope.com/events/vam23/

Event : BioEurope Fall 2023 (November 6-8, 2023; Munich, Germany) 

Gilles ALBERICI, CEO at ORPHELIA Pharma and Hugues BIENAYME, General Manager will attend the BioEurope Fall meeting to be held November 6-8, 2023 in Munich, Germany. BioEurope convenes over 5,500 attendees, representing 60 countries and 2,220+ companies, making the event the industry’s largest gathering of biopharma professionals in Europe.

We would be glad to meet you there and discuss partnering opportunities with ORPHELIA Pharma. Please contact us through the partnering portal at: https://informaconnect.com/bioeurope/pone/login/

Orphelia Pharma files EU marketing authorization application for KIZFIZO®

First drinkable formulation of temozolomide for treatment of relapsed or refractory neuroblastoma

Paris and Lyon, France, October 24, 2023 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, today announces the filing of a centralized Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for KIZFIZO®, the first oral liquid formulation of temozolomide.

KIZFIZO (temozolomide oral suspension, 40 mg/ml), known as Ped-TMZ or KIMOZO during its clinical development and ongoing early access programs, is designed specifically for use in the treatment of children with relapsed or refractory neuroblastoma, oncology indications with a very poor prognosis. This oral suspension, which is taste-masked, was developed for children : it allows a precise dose to be administered orally or via a nasogastric tube in a small volume. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last six years.

“We take great pride in having developed this new pediatric medicine,” said Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma. “KIZFIZO fills an unmet medical need for a drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma. This formulation aims to avoid the use of
non-age-appropriate dosage forms mixed with a drink or food, which may expose caregivers to a cytotoxic molecule without full control of the dose actually delivered to the patient.”

“The application for a drug marketing authorization of KIZFIZO is excellent news for the children living with cancer and their families. It underscores Gustave Roussy’s commitment to playing a leading role in the development of medicines for children and curing all of them in the future,” said Prof. Fabrice Barlesi, CEO of Gustave Roussy.
The pharmacokinetics of KIZFIZO in children have been evaluated in TEMOkids, a European multicenter population pharmacokinetic acceptability and safety study in pediatric patients in need of temozolomide (NCT04610736).

Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma submitted in the application includes in particular :

  • BEACON-Chemo, a sub-analysis of the chemotherapy arms of the BEACON study, a prospective randomized phase II study in refractory or relapsed neuroblastoma. This study was sponsored by Birmingham University (UK)
  • Retro-TMZ, a multicenter descriptive, retrospective study, assessing the efficacy and tolerability of temozolomide in children with refractory or relapsed neuroblastoma. This study was conducted by Gustave Roussy (France)

About KIZFIZO® 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume,
while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.
In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with irinotecan or topotecan.
KIZFIZO has received Orphan Drug Designation (ODD) from the EMA and the FDA, the formulation is covered by granted patents and pending applications in Europe and the US.

About neuroblastoma
Neuroblastoma is the most common extracranial cancer in early childhood, with approximately 900 new cases diagnosed per year in the European Union. It almost exclusively affects children under five, with a median age at diagnosis of 18 months. Neuroblastoma has a wide diversity of clinical outcomes, which is reflected in the risk stratification. Approximately 40% of patients have the high-risk disease and often face a poor response to first line induction therapy or later relapse. There remains a high unmet need for relapsed or refractory neuroblastoma patients and the best therapeutic strategy is still an intensive area of research. Temozolomide is the standard chemotherapy and is therefore an essential part of the treatment armamentarium for these patients.

About Orphelia Pharma
Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.
www.orphelia-pharma.eu

About Gustave Roussy
Ranked as the leading European Cancer Center and third in the world, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering from cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. It is a source of diagnostic and therapeutic advances. Per year, it caters for almost 50,000 patients, including 3,500 pediatric patients, and its approach is one that integrates research, patient care and teaching. It is specialized in the treatment of rare cancers and complex tumors and it treats all cancers in patients of any age. Its care is personalized and combines the most advanced medical methods with an appreciation of the patient’s human requirements. In addition to the quality of treatment offered, the physical, psychological and social aspects of the patient’s life are respected. 4,100 professionals work on its two campuses: Villejuif and Chevilly-Larue. Gustave Roussy brings together the skills which are essential for the highest quality research in oncology : 40% of patients treated are included in clinical studies.
For further information : www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram

Press and Analyst Contacts
Andrew Lloyd and Associates
Emilie Chouinard / Saffiyah Khalique
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ORPHELIA Pharma is at SIOP 2023 in Ottawa!

Stop by our booth in the exhibition and poster area to learn more about our pipeline including KIZFIZO, temozolomide oral suspension (product in development).

You are also invited to attend the oral session entitled “novel therapeutic approaches” on Saturday morning. Dr. Lucy METAYER, pediatric oncologist at Gustave Roussy, will present the results of the TEMOkids pediatric trial.

Orphelia Pharma to present results of the TEMOkids study (pharmacokinetic,acceptability and safety of KIZFIZO®) at the 2023 SIOP annual meeting

Paris and Lyon, France, October 4, 2023Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines announces today that results from the pediatric TEMOkids clinical trial will be presented at the 55th Société Internationale d’Oncologie Pédiatrique (SIOP) annual congress to be held in Ottawa, Canada, October 11-14, 2023. The results will be presented on October 14 during the oral session entitled “novel therapeutic approaches” by Dr. Lucy METAYER, co-investigator of the TEMOkids study and pediatric oncologist at Gustave Roussy, ranked 3rd cancer center worldwide (Villejuif, France).

TEMOkids (NCT04610736) is a European, multicenter, population pharmacokinetic, acceptability and safety clinical study evaluating KIZFIZO® (temozolomide oral suspension, 40 mg/ml) in children from the age of one year and in need of temozolomide. KIZFIZO, formerly known as KIMOZO or Ped-TMZ, has been designed specifically for use in the treatment of children with relapsed or refractory neuroblastoma, an oncology indication of dismal prognosis.

Forty-three young patients were recruited in TEMOkids across 12 European clinical cancer centers”, comments Caroline LEMARCHAND, Chief Pharmaceutical Development Officer at Orphelia Pharma. “TEMOkids is an integral part of the clinical development plan of KIZFIZO and results will be presented at the SIOP conference for the first time”.

Results from TEMOkids are very compelling” adds Dr. Samuel ABBOU, principal investigator of theTEMOkids study and pediatric oncologist at Gustave Roussy. “Not only the population pharmacokinetics show that there is no need for temozolomide dose adjustment in the pediatric population, but KIZFIZO was well accepted by children and its safety profile was similar to that of temozolomide capsules (Temodal), with no specific signal of local intolerance”.

About the TEMOkids study (NCT04610736)

TEMOkids is an international, open-label, non-randomized, study evaluating Ped-TMZ oral suspension (KIZFIZO) in 40 pediatric patients aged 1 year and over. The main objective of this study was to determine the pharmacokinetic parameters of KIZFIZO in this population. The secondary objectives were to evaluate its tolerance and acceptability by children, and their response to treatment.

Twelve clinical centers were involved in the TEMOkids study: Gustave Roussy, Villejuif, France (coordinating center); Institut Curie, Paris, France; La Timone Children’s Hospital, Marseille, France; Institute of Pediatric Hematology and Oncology, Lyon, France; Oscar Lambret Center, Lille, France; Charité University Medical Hospital, Berlin, Germany; Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands; Hospital Universitari Vall d’Hebron, Barcelona, Spain; Hospital La Fe, Valencia, Spain; University Pediatric Hospital Niño Jesús, Madrid, Spain; Southampton General Hospital, the United Kingdom and Great Ormond Street Hospital for Children NHS Trust, London, the United Kingdom.

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory neuroblastoma, the most common extracranial solid tumor of childhood carrying a dismal prognosis. This age-adapted and taste-masked formulation delivers an accurate drug load in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a fruitful collaboration between the pharmacists and clinicians of Gustave Roussy Cancer Center and the development team of Orphelia Pharma.

KIZFIZO, under the product name KIMOZO, has been granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities in March 2022 for the treatment of refractory and relapsed neuroblastoma.

KIZFIZO received orphan drug designation from the EMA and FDA and its formulation is covered by granted patents and pending applications in Europe and in the US.

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint on other continents.

For further information, please visit www.orphelia-pharma.eu.  

About Gustave Roussy

Ranked as the leading European Cancer Centre and third in the world, Gustave Roussy is a centre with comprehensive expertise and is devoted entirely to patients suffering with cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. It is a source of diagnostic and therapeutic advances. It caters for almost 50,000 patients per year and its approach is one that integrates research, patient care and teaching. It is specialized in the treatment of rare cancers and complex tumors and it treats all cancers in patients of any age. Its care is personalized and combines the most advanced medical methods with an appreciation of the patient’s human requirements. In addition to the quality of treatment offered, the physical, psychological and social aspects of the patient’s life are respected. 4,100 professionals work on its two campuses: Villejuif and Chevilly-Larue. Gustave Roussy brings together the skills, which are essential for the highest quality research in oncology: 40% of patients treated are included in clinical studies. 

For further information: www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram

About SIOP

The SIOP Annual Meetings bring together more than 2,500 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. This congress represents a unique opportunity to stay up to date in this rapidly moving field and to bring the latest science to bedside. The 55th annual meeting will be held in Ottawa, Canada, October 11-14, 2023.

Media Contact details

ORPHELIA Pharma : orphelia@orphelia-pharma.eu  / Tel : +33 (0)1 42 77 08 18

Event : SIOPEN annual general meeting, October 4-6 2023, Ljubljana, Slovenia

Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma will attend the SIOPEN annual general meeting to be held on October 4-6, 2023 in Ljubljana, Slovenia. The SIOPEN annual meeting brings together about 200 clinicians, investigators, researchers and patient advocates from 34 European countries to discuss on-going clinical trials, results, and work in progress in the field of neuroblastoma. If you would like to arrange a meeting with Jeremy on October 5 or 6, please contact him at: jeremy.bastid@orphelia-pharma.eu

Event : ANR 2023 (15-18 May, Amsterdam, the Netherlands) 

Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma will attend the Advances in Neuroblastoma Research (ANR) meeting to be held May 15-18, 2023 in Amsterdam, the Netherlands. 

ANR meetings gather investigators from around the world studying neuroblastoma biology, diagnosis, prognosis, and therapy.  

If you would like to arrange a meeting with Jeremy, please contact him at: jeremy.bastid@orphelia-pharma.eu  

Event : SIOPe (May 8-12, Valencia, Spain)

ORPHELIA Pharma will participate to the 4th Annual Meeting of the European Society for Paediatric Oncology (SIOPe 2023) to be held May 8-12, 2023 in Valencia, Spain. SIOPe aims to bring together the diverse stakeholders in a unique interactive format to discuss the current priorities and needs in the field of childhood cancers (https://siopeurope.eu/).

We are pleased to announce that Jeremy Bastid, Chief Development Officer, and Hugues Bienaymé, General Manager and Chief Scientific Officer at ORPHELIA Pharma, will attend SIOP Europe 2023. We will be present at booth #10 located near the entrance doors to Auditorium 1. Our booth will showcase the scientific data of our paediatric temozolomide oral suspension (KIMOZO).

If you would like to meet Jeremy or Hugues, please contact them at: jeremy.bastid@orphelia-pharma.eu or hugues.bienayme@orphelia-pharma.eu

KIMOZO 40 mg/ml is a ready-to-use and taste-masked oral suspension of temozolomide. KIMOZO is being developed by ORPHELIA Pharma for the treatment of relapsed or refractory neuroblastoma in pediatric patients aged 12 months and above. KIMOZO is not an approved medicinal product and is made available in certain European countries only via a named patient access program by ORPHELIA Pharma or Tanner Pharma (www.tannerpharma.com).

ORPHELIA Pharma selects Tanner Pharma to Initiate Named Patient Program for KIMOZO®

Paris (France) and Charlotte, N.C. (USA), on April 18th, 2023ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, and Tanner Pharma Group, a global provider of integrated specialty access solutions, have signed an exclusive agreement to manage the supply and distribution of Ped-TMZ, also known as KIMOZO® (temozolomide 40 mg/ml, oral suspension), to meet the special demands which may come from European physicians for this unapproved pediatric form of temozolomide.

Temozolomide is an anti-cancer drug approved as capsules or IV formulation in Europe for certain brain tumors. It is also used in treatment protocols for other rare cancer indications that mainly affect very young children, including refractory or relapsed neuroblastoma. In young children, an oral suspension of temozolomide is age-appropriate to ensure correct medication administration.

Under the terms of the agreement, Tanner Pharma will provide access to KIMOZO® via a named patient program in European countries, in particular these countries where KIMOZO® has already been used as investigational medication in the context of the now fully enrolled clinical trial TEMOkids sponsored by ORPHELIA Pharma. A named patient program is a mechanism through which physicians can legally and ethically prescribe investigational or approved drugs for patients prior to their commercial availability. The access to unapproved drugs is strictly regulated and adherence to national regulations is mandatory.

Tanner Pharma will manage all elements of the supply including healthcare practitioner enquiry management, national regulatory oversight, liaison with national authorities, and logistics. The agreement currently covers all European territories but France.

Hugues Bienaymé, General Manager of ORPHELIA Pharma, said:

KIMOZO® is currently available in France through an early access program, and ORPHELIA has already received requests from physicians from other countries. To meet these demands, ORPHELIA Pharma will use the services of Tanner Pharma, a world-leading pharmaceutical services provider with extensive experience in enabling access to innovative therapies when no other treatment options exist. With this agreement, our ambition is to make KIMOZO® available to children in need of a ready-to-use drinkable suspension of temozolomide, anywhere in Europe and with no delay, in strict adherence with local regulations.”

Rob Keel, Executive Vice President at Tanner Pharma, said:

“We are pleased to be partnering with ORPHELIA Pharma on this impactful program to help children with neuroblastoma and other malignancies. ORPHELIA Pharma has done extensive research to develop a product to address a critical unmet need for pediatric patients. We look forward to supporting physicians and enabling access to this innovative treatment.”

Healthcare professionals can obtain details about the products by contacting: kimozo@tannerpharma.com

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use drinkable formulation of temozolomide developed in collaboration between the pharmacists and clinicians of Gustave Roussy hospital and the development team of ORPHELIA Pharma. KIMOZO® is being developed for the treatment of refractory and relapsed neuroblastoma, a childhood cancer of dismal prognosis.

KIMOZO® has been granted Early Access Authorization (Autorisation d’Accès Précoce) for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with a specific DNA inhibitor topoisomerase I (irinotecan or topotecan) in patients aged 1 to 6 years and patients over 6 years of age unable to swallow temozolomide in capsule form, by the French authorities in March 2022.

The formulation of KIMOZO® has been covered by a patent application in Europe and in the US.

About ORPHELIA Pharma

ORPHELIA Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.

About Tanner Pharma

Tanner Pharma Group is a specialist pharmaceutical services provider based in Charlotte, USA with offices in Europe and Latin America. Over the past 20 years, Tanner has developed a portfolio of service offerings focused on improving global access to medicines. Through its TannerMAP division, Tanner Pharma supports manufacturers by providing ethical, controlled and compliant access to their innovative medicines in countries where they are not commercially accessible. For more information, visit www.tannerpharma.com.

Media Contact details

ORPHELIA Pharma : orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

Tanner Pharma Group : lstevens@tannerpharma.com / Tel: +1 704 552 8408

ORPHELIA Pharma announces completion of patient recruitment for the TEMOkids clinical trial

Paris and Lyon, March 2nd, 2023 – ORPHELIA Pharma, the French biopharmaceutical company that develops and markets pediatric medicines in the fields of oncology and neurology, announces the recruitment of the last patient of its TEMOkids clinical trial evaluating its ready-to-use temozolomide oral suspension, named KIMOZO® or Ped-TMZ, in children with a solid tumor from the age 1 year.
The TEMOKids trial, during which KIMOZO® was administered to children for the first time, is a major milestone in its clinical development. In addition to generating pharmacokinetic data, the trial also evaluates the safety and acceptability of the suspension in the study population and collects data on treatment response.
TEMOkids complements the study previously carried out in adults, which demonstrated the bioequivalence between KIMOZO® oral suspension and the capsule form of temozolomide, which is not adapted to children and yet regularly used in the absence of a pediatric form. ORPHELIA Pharma will present the results of the TEMOkids study at an upcoming scientific congress and will submit the marketing authorization dossier to the European Medicines Agency in 2023.

We have completed the enrollment of the TEMOkids clinical study thanks to the involvement and motivation of the investigating physicians, which underlines the medical need. With the support of a pediatric oncology network at the European level, we enrolled 43 children in 11 centers across 5 countries: France, Spain, the United Kingdom, the Netherlands and Germany“, says Caroline Lemarchand, Director of Pharmaceutical Development and coordinator of the TEMOkids study.

The inclusion of the last patient in TEMOkids is a key milestone of the clinical development of KIMOZO®. This new drinkable formulation of temozolomide addresses major medical needs for the management of cancers affecting young children,” concludes Jeremy Bastid, Director of Development at ORPHELIA Pharma. “Our goal is to make the product available to all patients as soon as possible. The ORPHELIA Pharma team is mobilized to enable the filing of the European registration dossier in 2023 and to fill the needs of young patients through special access programs, in close liaison with and with the approval of the national regulatory authorities in Europe,” he adds.

About the TEMOkids study
The clinical trial “TEMOkids Study: A Phase I Pediatric Study for KIMOZO®, Oral Suspension of temozolomide®” (NCT04610736) is an international, open-label, non-randomized Phase I study in 40 pediatric patients aged one year or older. The primary objective of the study is to evaluate the pharmacokinetics of KIMOZO®/Ped-TMZ in the study population. Secondary objectives are to assess tolerability and response to treatment, as well as acceptability in children. Eleven clinical centers are actively involved in the TEMOkids study: Gustave Roussy, Villejuif, France (coordinating center); Institut Curie, Paris, France; CHU Timone Enfants, Marseille, France; Institute of Hematology and Pediatric Oncology, Lyon, France; Centre Oscar Lambret, Lille, France; Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands; Hospital Universitari Vall d’Hebron, Barcelona, Spain; Hospital La Fe, Valencia, Spain; University Pediatric Hospital Niño Jesús, Madrid, Spain; Southampton General Hospital, Southampton, United Kingdom; Great Ormond Street Hospital (GOSH) for Children NHS Trust, London, United Kingdom; Charité University Medicine Hospital, Berlin, Germany.
For more information, please visit www.temokids.eu.

About KIMOZO®/Ped-TMZ oral suspension, 40 mg/ml
KIMOZO® 40 mg/ml is a ready-to-use, taste-masked oral liquid formulation of temozolomide jointly developed by the ORPHELIA Pharma development team and the pharmacists and clinicians of Gustave Roussy for the treatment of relapsed or refractory neuroblastoma, a childhood cancer with a poor prognosis.
The product obtained an Early Access Authorization from the French authorities in March 2022.

About ORPHELIA Pharma
ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of rare diseases, with formulations adapted to the pediatric population. With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.
More information on www.orphelia-pharma.eu.

Contacts : orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

Event : BIO-EUROPE 2022 (Oct. 24-26, Leipzig, Germany)

ORPHELIA Pharma will participate to BIO-EUROPE 2022 to be held from October 24-26, 2022 in Leipzig, Germany. This annual meeting aims to gather and connect biotechnology, pharmaceutical industry, and financial sector on pharmaceutical innovative projects.

We are glad to announce that Gilles Alberici, Executive Chairman and Hugues Bienaymé, General Manager and Chief Scientific Officer at ORPHELIA Pharma, will attend this event. If you would like to meet Gilles and Hugues, please contact them : contact@orphelia-pharma.eu.  

Sponsored by : La Région Auvergne – Rhône – Alpes

Publication: dispensing temozolomide using KIMOZO or Temodal capsules mixed with food

ORPHELIA Pharma, the French pharmaceutical company that develops and commercializes orphan and pediatric medicines in the fields of oncology and neurology, announces the publication of the results of its study performed in collaboration with the Pharmacy Department of Gustave Roussy and evaluating the temozolomide dose actually delivered to children when using, on the one hand, KIMOZO oral suspension and, on the other hand, Temodal capsule content mixed with soft food or drinks.

The poster #128  entitled “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures” (Lemarchand et al.), is displayed during the poster session of the SIOP 2022 Annual Congress held in Barcelona (Spain). The poster can also be accessed by using this link.

About SIOP

SIOP (International Society of Pediatric Oncology) is the only multidisciplinary and international learned society entirely dedicated to childhood and adolescent cancer. The society has more than 2,600 members worldwide, including doctors, nurses, other health professionals, scientists and researchers. Members are dedicated to increasing knowledge about all aspects of childhood cancer (https://siop-online.org/). The annual congress is held in Barcelona, Spain, from 28th September to 1st October, 2022 (https://siop-congress.org/).

SIOP 2022, Barcelona, Spain : September 28-October 1, 2022

ORPHELIA Pharma is proud to announce that it will attend 54th Annual SIOP Congress in Barcelona, Spain. M. Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma, will have the pleasure to present a poster entitled: “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures”.

The SIOP 2022 Annual Meetings will bring together more than 3,000 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. The SIOP 2020 Annual Meeting will take place in Barcelona, Spain, from September 28-October 1, 2022 (https://siop-congress.org/). 

Hugues Bienaymé, General Manager, and Jeremy Bastid, Chief Development Officer at OPRHELIA Pharma, will attend this event. If you would like to meet with Hugues and Jeremy, please send an email to: contact@orphelia-pharma.eu.