ORPHELIA Pharma and Ethypharm sign a licensing agreement for the development and marketing of Kigabeq® in China

Saint-Cloud and Paris – January 19, 2021 – Ethypharm and ORPHELIA Pharma announce today the signature of an exclusive agreement for the development, registration and marketing in the People’s Republic of China of Kigabeq® (vigabatrin) indicated for the first-line treatment of infantile spasms (West syndrome).

 Kigabeq®, the first paediatric form of vigabatrin developed by ORPHELIA Pharma, is approved and marketed in Europe, where this medicine, intended exclusively for children, has been granted a Paediatric Use Marketing Authorisation (PUMA). Kigabeq® is indicated for the first-line treatment of infantile spasms (West’s syndrome), a severe epileptic encephalopathy in infants.

There is a significant unmet medical need in China for patients suffering from this rare and devastating form of epilepsy. By entering into this agreement, Ethypharm and ORPHELIA Pharma are committed to providing appropriate treatment for Chinese children suffering from this rare disease.

As part of this agreement, ORPHELIA Pharma grants Ethypharm exclusive rights to Kigabeq® in the People’s Republic of China. After registration, Ethypharm will be responsible for the distribution and sale of Kigabeq® in China.

 “Our objective is to make Kigabeq® available in many territories. After Europe and China, new agreements will be signed to enable the marketing of this formulation of vigabatrin, which is particularly suitable for children,” comments Hugues BIENAYME, founder and Managing Director of ORPHELIA Pharma.

 “We are very pleased to establish this partnership with ORPHELIA Pharma. This collaboration brings an effective solution to young Chinese patients. The upcoming commercialisation of Kigabeq® is fully in line with our strategy to strengthen our rare disease franchise in China” said Bertrand DELUARD, President and CEO of Ethypharm.

 Through this partnership, Ethypharm is strengthening its ambition to become a key player in the field of rare diseases in China. Operating successfully for more than 20 years in an ever-changing Chinese environment, Ethypharm has a fully integrated subsidiary located in Shanghai. “Our subsidiary is a partner of choice for companies wishing to make their existing internationally-marketed orphan drugs or drugs for rare diseases available to Chinese patients,” says François LIOT, General manager of Shanghai Ethypharm.

“I am delighted to sign this agreement with Ethypharm, which is based on the value of Kigabeq® in paediatrics. Thanks to the in-depth knowledge that the Ethypharm Group is developing in China, we are very confident that chinese children suffering from infantile spasms will have access to Kigabeq® as quickly as possible,” adds Gilles ALBERICI, Chairman of ORPHELIA Pharma.

About Kigabeq®

Kigabeq® is the first paediatric formulation of vigabatrin, an essential anti-epileptic drug. Kigabeq® is available in 100 mg and 500 mg soluble and scored tablets for oral or (naso)gastric administration and is indicated for the first-line treatment of childhood spasms (West’s syndrome), a severe epileptic encephalopathy in infants. Kigabeq® has been developed exclusively for children and has been granted Paediatric Use Marketing Authorisation (PUMA).

About infantile spasms

West’s syndrome, or infantile spasms, is a severe epileptic encephalopathy of the infant that combines epileptic spasms, a psychomotor deterioration and an hypsarrhythmic electroencephalogram tracing. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in infants with previously normal development or with a pre-existing delay; in all cases, infantile spasms have a strong impact on the psychomotor development. Pharmacological treatment must be started quickly to allow the spasms to stop and improve the prognosis.

 About the Ethypharm Group

Ethypharm is a European pharmaceutical company focused on two therapeutic areas: the Central Nervous System and Critical Care. Ethypharm markets its drugs directly in Europe and China, and with partners in North America and the Middle East where its drugs are in high demand. The Group employs more than 1,500 people, mainly in Europe and China.

Ethypharm works closely with authorities and healthcare professionals to ensure the appropriate use of and access to its medicines, by as many people as possible.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorisation in September 2018 and is being launched in several countries. Our second product, Ivozall®, obtained European marketing authorisation in November 2019 for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.

Press contact

Avril PONNELLE / presse@ethypharm.com / + 33 (0)1 41 12 17 20

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

Paris, October 12^th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs announces today that it will organize a symposium at the Société Internationale d’Oncologie Pédiatrique (SIOP) congress 2020. The session will focus on the pediatric use of temozolomide in the treatment of relapsed or refractory pediatric tumors and will showcase the development of Kimozo a ready-to-use and taste-masked oral suspension of temozolomide adapted to children.

“ORPHELIA Pharma is committed to improving the care of patients with pediatric and rare diseases, including developing innovative formulations to address children needs,” comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “Our commitment to develop child-friendly medicines is a historical pillar of our company and this session at the SIOP meeting highlights our long-term close collaboration with the pediatric oncology community”.

This industry symposium entitled “Improving children care with temozolomide oral suspension” will be held virtually on Friday, October 16^th from 9:15 AM to 9:35 AM local time (Ottawa), from 3:15 PM. to 3:35 PM. Central European Time (CET) and accessible by connecting on the SIOP meeting website (SIOP-congress.eu) in the “Product theatre” section.

Dr Lucas Moreno (Val d’Hebron Hospital, Spain) and Dr Dominique Valteau-Couanet (Gustave Roussy Hospital, France), two internationally renowned clinicians, will talk about the critical role of temozolomide in the management of relapsed or refractory pediatric tumors, including high-risk neuroblastomas. The third presenter, Dr Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma, will discuss the development of Kimozo, the first pediatric formulation of temozolomide under clinical development.

We look forward to welcoming you during this unique event.

About Kimozo

Kimozo (also known as Ped-TMZ or ORP-005) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under clinical development to address children needs. Kimozo is an investigational medicinal product not yet approved for use or marketing anywhere in the world.

About SIOP 2020 virtual congress

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 an unique opportunity to stay up to date in this rapidly moving field and to bring the latest science to bedside. In light of the impact of the COVID-19 pandemic on travel restrictions and physical distancing requirements, the SIOP 2020 Annual Meeting will be held virtually on October 14-17, 2020.

ORPHELIA announces the inclusion of the first patient in a clinical study evaluating Kimozo, its pediatric formulation of temozolomide

Paris and Lyon, October 5^th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs is pleased to announce the inclusion of the first-in patient in a clinical study aimed at demonstrating the bioequivalence between Kimozo, the first pediatric formulation of temozolomide under clinical development, and Temodal® capsule.

Although not approved in this indication, temozolomide is an essential anticancer medicine recommended for use in treatment protocols of relapsed or refractory neuroblastoma, a condition that affects young children. However, the only oral formulations that are commercially available are in the form of capsules that are poorly adapted to children. Caretakers are thus compelled to open capsules and disperse the content into a soft food prior to administration to the child, which harbors several risks such as the uncertainty with regards to the delivered dose, the exposure of the caregiver to the cytotoxic drug and the instability of the drug in aqueous milieu. To overcome the risks this situation implies, Kimozo, a ready to use oral formulation has been specifically designed to address the needs of the pediatric population.

Kimozo has been developed in collaboration with Gustave Roussy, one of the leading cancer centers in Europe. The current clinical trial conducted by ORPHELIA Pharma aims at demonstrating, first for regulatory purposes, the bioequivalence between Kimozo and the Temodal® capsules in adult patients having brain cancers, with additional clinical investigation to come in the pediatric population of interest.

“We have reached an important milestone for Kimozo with the clinical trial approval from competent authorities and the recruitment of a first patient“, said Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma “We plan to enroll 30 patients by mid-2021 thanks to the support of the three neuro-oncology teams involved in the study: Professor Ducray of the Hospices Civils de Lyon (coordinating investigator), Professor Chinot of the Timone Hospital in Marseille and Dr Bronnimann of Saint-André Hospital in Bordeaux. ”

“We are pleased to contribute to the development of this new pediatric formulation of temozolomide. A liquid form is unambiguously of interest for treating children.“, underlines Pr. François Ducray. Hugues Bienaymé, General Manager of ORPHELIA Pharma further comments: “This first administration is a major milestone in the development of Kimozo. We are now preparing the opening of our second clinical trial, which will evaluate Kimozo in pediatric patients, by the end of the year ”, he concludes.

About the Bioequivalence Study (NCT04467346)

The clinical trial entitled “Bioequivalence Study between temozolomide oral suspension (Ped-TMZ) and Temodal® capsules” (NCT04467346) is an open label phase I study, randomized, crossover, 2-period study in 30 male/female patients with primary CNS malignancies. Patients will receive, under fasting conditions, 200 mg/m² of Temozolomide Oral Suspension (Ped-TMZ, the code name of Kimozo) or Temodal®, as single oral administration in 2 different study periods depending on the randomization, with no wash out period between administrations. The primary objective is to evaluate the bioequivalence between Kimozo and Temodal® capsules for oral administration. The secondary objectives are to define the pharmacokinetic parameters of Kimozo administration and to assess its buccal safety.

The clinical centers are Hospices Civils de Lyon, Bron, France ; CHU de Bordeaux, Bordeaux, France and Hôpital de la Timone (AP-HM), Marseille, France. The Study sponsor is ORPHELIA Pharma.

About Kimozo

Kimozo (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address children needs. Kimozo is an investigational medicinal product not yet approved for marketing anywhere in the world.

Meet us at BIO-Europe 2020, October 26-29

Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will participate in the upcoming BIO-Europe conference to be held virtuallly on october 26-29, 2020. BIO-Europe is Europe’s largest partnering conference serving the global biotech industry.

Jeremy will actively take part in this business event, registered under the name of the holding company, initiative OCTALFA.

ORPHELIA Pharma will support and attend the upcoming SIOP Congress from October 14^th to 17^th (52^nd Congress of the International Society of Paediatric Oncology), which will be virtual this year.

This event aims to bring together the oncology community and share the latest advances in pediatric oncology.

Join the ORPHELIA Pharma virtual symposium to share on innovation in the use of temozolomide in children, which will take place on Friday October 16^th between 9:15 AM and 9:35 AM (Ottawa time) by logging on to the SIOP website (SIOP-Congress.org).

Please contact us if you have any questions (pierre-julien.cotte@orphelia-pharma.eu).

Paris and Lyon, March 11th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces the appointment of Laurent Martin as Chief Pharmaceutical Affairs Officer, effective January 6^th, 2020.

Pharmacist and scientist by background, Laurent has got more than 25 years of experience in drug development, especially in rare and serious pathologies. Laurent contributed to the development, the European approval and the marketing of several orphan drugs at Orphan Europe (now Recordati Rare Diseases). More recently, he was Chief Development Officer and Qualified Person at DBV Technologies, where he led global developments (USA, Europe) of innovative biological drugs mainly for children.

« Laurent’s large experience in the development of drugs for rare and paediatric diseases is a significant asset for ORPHELIA Pharma » comments Hugues Bienaymé, Founder and General Manager. « Laurent will be instrumental in strengthening ORPHELIA Pharma’s pharmaceutical structure. We plan to file an application to operate as a pharmaceutical licensed company (the French “Exploitant” status) in order to market our products in France. »

« I was convinced by ORPHELIA Pharma’s model, which I am very happy to join at this period of strong development of the company » says Laurent Martin. « The essential paediatric products developed by ORPHELIA Pharma perfectly match with my regulatory expertise and my wish to work on the marketing of medicines with high medical benefit.»

ORPHELIA Pharma raises 4 m€ to fund its commercial operations and clinical developments

French family offices join as new shareholders and Board members

Paris and Lyon, January 28^th , 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces it has raised 4 million € in December 2019. The proceeds will be used to grow the company’s team, develop its commercial and clinical infrastructure and advance its projects.

Initiative OCTALFA led this financing round and was joined by three French family offices managed by serial entrepreneurs, namely Ravenala holding (Alain Tornier), PAF Kapital (Christophe Pasik, Jean-François Auffret and Pierre Ferran) and Cemag (André Ulmann).

« We thank all our shareholders for their confidence in our venture and our programs », said Hugues Bienaymé, founder and CEO of ORPHELIA Pharma. « In 2020, we plan to record our first revenues with the launch of Kigabeq® and Ivozall® and initiate two clinical trials. We will also apply for a pharmaceutical establishment license in France and we anticipate to hire 7 new people. »

In addition, the company announced that André Ulmann and Christophe Pasik have joined ORPHELIA Pharma’s Board of Directors.

« We are particularly pleased to have Alain, André and Christophe with us. Their track record as successful entrepreneurs will be of invaluable help in guiding ORPHELIA Pharma to building a portfolio of paediatric medicines for rare and severe diseases » added Gilles Alberici, Chairman of ORPHELIA Pharma. « We look forward to working with them to accelerate the structuring of the company and to develop and launch new effective medicines for children. »

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, obtained its Marketing Authorization in September 2018 and is being launched in Europe. Our second product, Ivozall®, was granted a European marketing authorization in November 2019 for the treatment of Acute lymphoblastic Leukaemia. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations.

ORPHELIA Pharma announces the commercial launch of Kigabeq® in France

Paris and Lyon, January 7^th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces today the preparation of Kigabeq® launch in France after having reached an agreement with the Comité Economique des Produits de Santé (CEPS).

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 was granted a Paediatric Use Marketing Authorization (PUMA).

« The price fixing in France highlights the clinical significance of Kigabeq® from the Haute Autorité de Santé (HAS) and CEPS perspectives, is the ultimate step prior to effective market launch in France », comments Hugues Bienaymé, general Manager of ORPHELIA Pharma. « We are working hard with our manufacturer and our wholesaler to make Kigabeq® available to neuropediatricians from April 2020 ».

« Vigabatrin is an essential component of the treatment of infantile spasms or West syndrome », adds Pr. Stéphane Auvin, neuropediatrician at Robert Debré hospital. « For this population of very young children, the accuracy of delivered dose and the ease of use allowed by Kigabeq® will facilitate therapeutic management ».

« The upcoming launch of Kigabeq® is the culmination of several years of research and efforts carried out in collaboration with ORPHELIA Pharma » concludes Dr. Catherine Chiron, neuropediatrician at Necker-Enfants-Malades Hospital and Research Director at Inserm. « We are particularly pleased that this collaboration now provides an optimized solution to our young patients ».

About infantile spasms

West Syndrome, or infantile spasms, is an early childhood epileptic encephalopathy which combines axial spasms in cluster, a marked psychomotor retardation and an hypsarrhythmic electroencephalogram. It is a rare disease with an estimated incidence of around 5 per 10,000 living births. It affects infants whose development was normal or children with impaired cognitive development prior to spasms. Children with infantile spasms must be treated as soon as possible to stop spasms and improve prognosis.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, obtained a Marketing Authorization in September 2018 and is being launched in Europe. Our second product, Ivozall®, was granted a European Marketing Authorization in November 2019. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations.

ORPHELIA Pharma will be present in Toulouse on January, 15th to 17th, for the SFNP Congress (Société Française de Neuropédiatrie). This event aims to bring together the French community of pediatric neurologists and share latest advances in paediatric neurology. Do not hesitate to contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu)

ORPHELIA Pharma announces Orphan Drug Designation granted by FDA for temozolomide in the treatment of neuroblastoma

Paris and Lyon, December 10^th, 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology, announces today that the Office for Orphan Products of the Food and Drug Administration (FDA) has issued a positive opinion for the Orphan Drug Designation of temozolomide in the treatment of neuroblastoma.

« We are particularly pleased with the grant of this Orphan Designation », comments Jérémy Bastid, Chief Development Officer of ORPHELIA Pharma. « Temozolomide is an important part of the treatment armamentarium for high-risk neuroblastoma patients who relapse or face disease recurrence, albeit used off‑label. Existing formulations of temozolomide-containing products are not adapted to young children. Our formulation Kimozo® aims at providing these young patients with a pharmaceutical form adapted to their age », he concludes.

Kimozo® is the first presentation of the anticancer drug temozolomide developed for the treatment of relapsed or refractory neuroblastoma, a pediatric disease affecting young patients with dismal prognosis. Kimozo® is being developed as a taste-masked oral suspension and will be the first pediatric formulation of temozolomide.

« Temozolomide has become an integral component of established regimens for relapsed and refractory neuroblastoma. Patients with neuroblastoma are usually younger than 5 years of age and unable to tolerate the currently available temozolomide tablet formulations, presenting barriers to administration. The liquid formulation of Kimozo® is therefore a welcome and needed resource for treating patients with neuroblastoma », highlights Dr. Julie Park, Department of Pediatrics, Seattle Children’s Hospital and University of Washington School of Medicine, Seattle.

« The Office for Orphan Products of the FDA has acknowledged the benefit of temozolomide in the treatment of neuroblastoma patients with relapsed of refractory disease. The pediatric formulation that we are developing addresses significant unmet medical needs for young children with neuroblastoma », comments  Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « We expect to file Kimozo® Marketing Authorization Application with the FDA as soon as the dossier is complete ».

About temozolomide in neuroblastoma

Neuroblastoma is the most frequent extra-cranial solid tumor in children. It is a rare disease with an incidence of 1.3/100,000. Prognosis is extremely variable, from spontaneously regressing tumors in low-risk neuroblastomas to highly aggressive disease with dismal prognosis in high-risk patients. Half of neuroblastomas are classified as high-risk, among which half being refractory to treatment or relapsing thereafter. Temozolomide has become the mainstay of rescue treatment of relapsed or refractory neuroblastoma, without being authorized in this condition.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon (France) that develops and markets drugs for the treatment of rare and serious pediatric diseases. Its mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the pediatric population. Its first product, Kigabeq®, obtained its Marketing Authorization in Europe in October 2018 and has been launched in Europe. Its second product, Ivozall®, has recently been granted approval by the EMA for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

ORPHELIA Pharma announces the granting of the Marketing Authorisation for Ivozall® from the European Commission

Paris and Lyon, November 26^th, 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces today that the European Commission has granted Ivozall® an European Marketing Authorisation (MA).

Ivozall® is a solution for infusion containing 1 mg/ml of clofarabine and is supplied in 20 ml-vials. Clofarabine is an essential medicine for the treatment of relapsed or refractory Acute Lymphoblastic Leukaemia (ALL) in children. Ivozall® will be the first generic form of clofarabine to be authorised under the European centralized procedure.

« This approval is a significant milestone for ORPHELIA Pharma as Ivozall® is the first medicine in a series of oncology products we plan to launch. We believe it addresses important pediatric needs and will meaningfully improve children’s care », comments Jeremy Bastid, Chief Development Officer. « We will now focus on launching Ivozall® throughout Europe and building relationships with distribution partners to bring Ivozall® to patients with ALL ».

« This MA will allow the company to strengthen its product portfolio with this first oncology medicine in which we have great expectations » says Hugues Bienaymé, Founder and General Manager. « We anticipate to make Ivozall® available to hospital centres treating ALL patients in some countries from March 2020. Other countries will be handled by ORPHELIA’s distributors, to be selected in the course of 2020 ».

About Acute Lymphoblastic Leukaemia

ALL is a cancer associated with the uncontrolled proliferation of lymphoblasts that invade the bone marrow. The disease progresses rapidly and aggressively and requires immediate treatment. ALL is a rare disease, with around 7,000 people diagnosed each year in Europe. The majority of ALL cases occurs in children. Although rare, ALL is the most common type of childhood cancer. Clofarabine is indicated for the treatment of ALL in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population.

Our first product, Kigabeq®, obtained its MA in September 2018 and is being launched in Europe. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

Caroline Lemarchand, Pharmaceutical Development Director of ORPHELIA Pharma will be present to the 2019 CPhI to be held in Frankfurt (Germany), November 5 – 7, 2019.

Please meet us there (contact: caroline.lemarchand@orphelia-pharma.eu).

ORPHELIA Pharma and Veriton Pharma execute a license and distribution agreement for Kigabeq^® in the United Kingdom

Paris/Lyon and Weybridge, October 29^th, 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology, announces today that it has entered into a license and distribution agreement with Veriton Pharma to market Kigabeq^® in the United Kingdom.

Kigabeq^® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug, available as scored 100 mg and 500 mg soluble tablets for oral and (naso)gastric administration. Kigabeq^® has been developed exclusively for children aged between 1 month and 6 years and was granted a Paediatric Use Marketing Authorization (PUMA) in September 2018. Kigabeq^® is indicated in the first-line treatment for infantile spasms, an extremely severe early childhood encephalopathic epilepsy.

« As a renowned specialist in the treatment of epilepsy, Veriton Pharma is an outstanding partner for Kigabeq^® in the United Kingdom » comments Hugues Bienaymé, founder and General Manager of ORPHELIA Pharma. « After Germany, the UK is the second country where Kigabeq® will be offered to patients. We are now actively preparing launch of Kigabeq® in France and in other European countries with dedicated distribution partners. This new agreement confirms the continued attractiveness for Kigabeq® in Europe ».

« At Veriton Pharma, we are dedicated to providing tailored therapy options for people with special medical needs. We are excited to be the exclusive partner working alongside ORPHELIA in the UK and this collaboration aligns with Veriton’s core strategy to continuously deliver products to help support UK healthcare.», says Chris Grimes, Chief Commercial Officer of Veriton.

« This agreement with Veriton Pharma is an important step forward for Kigabeq®, our first product to receive EU approval and to reach the market. Kigabeq® addresses a significant unmet medical need in a rare and potentially devastating form of epilepsy and it aims at improving the life of children affected by this condition » adds Gilles Alberici, Chairman of ORPHELIA Pharma.

About infantile spasms

West Syndrome, or infantile spasms, is an early childhood epileptic encephalopathy which combines axial spasms in cluster, a marked psychomotor retardation and an hypsarrhythmic electroencephalogram. It is a rare disease with an estimated incidence of around 5 per 10,000 living births. It affects infants whose development was normal or children with impaired cognitive development prior to spasms. Children with infantile spasms must be treated as soon as possible to stop spasms and improve prognosis.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population.

Our first product, Kigabeq®, obtained its Marketing Authorization in October 2018 and is being launched in Europe. Our second product, Ivozall®, received a positive opinion from the CHMP in September 2019 recommending the issuance of a European marketing authorization. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

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

About Veriton Pharma

Veriton Pharma is a private pharmaceutical company founded in 1997. The company is based in Weybridge UK with regional offices in The Middle East and Australia where it supplies products to over 25 countries worldwide. Veriton Pharma focuses on the development of licensed medicines, predominantly for CNS, and unlicensed medicines for patients with special requirements that licensed products cannot meet.

The company has a portfolio of more than 60 specialised formulations for treating conditions across a range of therapeutic areas. Veriton Pharma’s products are supported in the UK by a team of Regional Account Managers who are able to access the NHS at all critical levels. Similarly, both the MEA and Australian offices are able to provide experienced sales support within each of these areas.

For more information, please visit www.veritonpharma.com

ORPHELIA Pharma will be present in Paris on November, 5th and 6th, for the Rare Diseases Meeting Days. This event is organized by the French Foundation for Rare Diseases every other year, and allows to assist during two days to plenary conferences and scientific round tables on current topics, poster presentations and meetings with researchers, meeting with companies and stakeholders of the sector.  

The organizing committee of the Foundation selected ORPHELIA’s poster entitled “Multicentre retrospective study of temozolomide use in paediatric refractory or relapsed neuroblastoma : Results from analysis of data from French centres”, to be presented during the Rare Diseases Meeting Days.

Do not hesitate to contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu).

ORPHELIA Pharma will be present in Lyon on October, 23th to 26th, for the annual SIOP Congress (the 51st Congress of the International Society for Paediatric Oncology). This event aims to bring together the oncology community and share latest advances in paediatric oncology. Do not hesitate to contact us in order to meet at this occasion (jeremy.bastid@orphelia-pharma.eu).

ORPHELIA announces the appointment of Dr. Caroline Lemarchand as Chief Pharmaceutical Development Officer

Paris and Lyon, October 15^th 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces the appointment of Dr. Caroline Lemarchand as Chief Pharmaceutical Development Officer, effective September 16, 2019.

Caroline Lemarchand, PharmD, PhD, will bring to ORPHELIA Pharma her expertise in pharmaceutical development, acquired during a 20-year career in pharma and biotech companies. Caroline was successively working at Onxeo (formerly Bioalliance Pharma) as preclinical and pharmaceutical development Director before joining Stallergenes Greer as pharmaceutical development Director.

Caroline Lemarchand contributed to the development, registration and marketing of several drugs (innovative technologies, new chemical entities, biotherapy), especially in the field of oncology, both in Europe and in the United States. She authored numerous peer-reviewed scientific publications and patents.

« We are particularly pleased to welcome Caroline at ORPHELIA Pharma » comments Hugues Bienaymé, Founder and General Manager at ORPHELIA Pharma. « Her deep knowledge of pharmaceutical development and impressive track-record will be a significant asset for a company like ORPHELIA Pharma, which focuses on life-threatening paediatric conditions. Caroline will be instrumental in managing our collaborative partnerships to assemble a portfolio of essential drugs for children ».

 « I am excited to be joining ORPHELIA Pharma at this important inflection point with already 2 products approved and several collaborative projects running », says Caroline Lemarchand. « The company is in a privileged position to build a portfolio of drugs for the paediatric community, both in neurology and oncology. Since there are many collaborations already in place, I look forward to leading these partnerships and setting up some new projects ».

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, obtained its Marketing Authorization in October 2018 and has been launched in Europe. Our second product, Ivozall®, received a positive opinion from the CHMP in September 2019 recommending the issuance of a European marketing authorization. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

ORPHELIA Pharma announces that the CHMP has adopted a positive opinion for Ivozall® (clofarabine 1 mg/ml), an essential medicine intended for the treatment of relapsed or refractory Acute Lymphoblastic Leukaemia in children

Paris and Lyon, October 1^st, 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the issuance of a marketing authorization for Ivozall®.

Ivozall® is a solution for infusion containing 1 mg/ml clofarabine and supplied in 20 ml-vials. Clofarabine is an essential medicine for the treatment of relapsed or refractory Acute Lymphoblastic Leukaemia (ALL) in children. Ivozall® will be the first generic form of clofarabine to be authorised under the European centralized procedure.

« We are particularly pleased with this decision which comes after continuous efforts of our company together with our partners », comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. « This positive opinion is a key milestone in the process of making Ivozall®, our first medicinal product in oncology, available to the paediatric community ».

« The CHMP has acknowledged the interest of Ivozall® in improving health of children with ALL. We are eagerly awaiting the marketing authorisation expected for November 2019 », highlights Hugues Bienaymé, General Manager of ORPHELIA Pharma. « We anticipate to make Ivozall® available to the hospital centres treating ALL patients in some countries from February 2020 », added Gilles Alberici, Chairman of ORPHELIA Pharma. « Other countries will be handled by ORPHELIA distributors, to be selected in the course of 2020 ».

About Acute Lymphoblastic Leukaemia

Acute Lymphoblastic Leukaemia is a cancer associated with the uncontrolled proliferation of lymphoblasts that invade the bone marrow. The disease progresses rapidly and aggressively and requires immediate treatment. ALL is a rare disease, with around 7,000 people diagnosed each year in Europe. The majority of ALL cases occurs in children. Although rare, ALL is the most common type of childhood cancer. Clofarabine is indicated for the treatment of ALL in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology using formulations adapted to the paediatric population. ORPHELIA Pharma conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

ORPHELIA Pharma will be present in Avignon on October, 9th to 11th, for the XIIth National Oncology Days of the French Society of Pharmacy Oncology. This event aims to bring together the pharmaceutical oncology community and inform them of advances in oncology treatments. Do not hesitate to contact us at this occasion (jeremy.bastid@orphelia-pharma.eu) and visit our exhibition booth at the “Palais des papes” in Avignon (booth 47 – “Paneterie” room).

ORPHELIA Pharma announces Orphan Drug Designation granted by EMA for temozolomide in the treatment of neuroblastoma

Development of Kimozo®, the first paediatric formulation of temozolomide

 Paris and Lyon, September 3^rd, 2019 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces today that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion for the Orphan Drug Designation of temozolomide in the treatment of neuroblastoma.

« We are particularly pleased with this decision which comes after continuous efforts of ORPHELIA Pharma together with its partners », comments Jérémy Bastid, Chief Development Officer. « We are developing Kimozo®, the first paediatric formulation of temozolomide, to provide patients and the medical community with a pharmaceutical form adapted to young children. This positive opinion is one step closer to this goal ».

Kimozo® is the first presentation of the anticancer drug temozolomide developed for the treatment of relapsed or refractory neuroblastoma, a paediatric disease with dismal prognosis. As a taste-masked oral suspension, it will also be the first paediatric formulation of temozolomide.

« Temozolomide has been used for years in the treatment of refractory or relapsed neuroblastoma. It is now considered to be an essential drug in this indication », highlights Dominique Valteau-Couanet, President of the Société Internationale d’Oncologie Pédiatrique, Europe Neuroblastoma (SIOPEN), « The liquid formulation of Kimozo®, which will be the only temozolomide-containing drug approved for the treatment of neuroblastoma, is particularly well adapted to the intended paediatric population since 90% of the patients are less than 5 years at diagnosis ».

« The COMP has acknowledged the benefit of temozolomide in the treatment of children with high-risk neuroblastoma. The paediatric formulation that we are developing addresses significant unmet medical needs for young children with neuroblastoma », comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « In Europe, we expect to submit Kimozo® Marketing Authorisation application file by 2021 ».

About temozolomide in neuroblastoma

Neuroblastoma is the most frequent extra-cranial solid tumour in children. It is a rare disease with an incidence of 1.3/100,000. Prognosis is extremely variable, from spontaneously regressing tumours in low-risk neuroblastomas to highly aggressive disease with dismal prognosis in high-risk patients. Half of neuroblastomas are classified as high-risk, among which half being refractory to treatment or relapsing thereafter. Temozolomide has become the mainstay of rescue treatment of relapsed or refractory neuroblastoma, without being authorized in this condition.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, obtained its Marketing Authorization in October 2018 and has been launched in Europe. Our second product, Ivozall®, is under review by the EMA for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.

ORPHELIA Pharma will be present in Paris on October, 7th to 10th, for the 22nd French Epilepsy Days. This congress is schedule by the “Ligue Française contre l’Épilepsie “ (LFCE), French subsidiary of the International Ligue Against Epilepsy (ILAE). Do not hesitate to contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu).