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

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 attend and support the annual SIOP Congress 2020

ORPHELIA Pharma will support and attend the upcoming SIOP Congress from October 14th to 17th (52nd 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 16th 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).

Appointment of L. Martin as Chief Pharmaceutical Affairs Officer

Paris and Lyon, March 11th, 2020ORPHELIA 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 6th, 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 fund raising

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 28th , 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.

Launch of Kigabeq® in France

ORPHELIA Pharma announces the commercial launch of Kigabeq® in France

Paris and Lyon, January 7th, 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.

ODD for temozolomide in the treatment of neuroblastoma

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

Paris and Lyon, December 10th, 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.

Granting of the Marketing Authorisation for Ivozall

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

Paris and Lyon, November 26th, 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.

ORPHELIA Pharma will be present at CPhI in Frankfurt

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).

License and distribution agreement for Kigabeq in the United Kingdom

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

Paris/Lyon and Weybridge, October 29th, 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 at the Rare Diseases Meeting Days

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 at the annual SIOP Congress

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).

C.Lemarchand – Chief Pharmaceutical Development Officer

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

Paris and Lyon, October 15th 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.

CHMP has adopted a positive opinion for Ivozall®

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 1st, 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 at the XIIth National Oncology Days

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).

ODD-EU for temozolomide in the treatment of neuroblastoma

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 3rd, 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 at the 22nd French Epilepsy Days

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).

ORPHELIA recrute un Responsable Affaires Règlementaires

Recrutement d’un Pharmacien(ne) en charge des Affaires Règlementaires :

De formation supérieure (Docteur en Pharmacie) avec une spécialisation en Affaires Réglementaires, vous justifiez d’une expérience d’au moins 3 ans sur une fonction similaire dans un laboratoire pharmaceutique. Vous disposez d’une bonne connaissance des réglementations Eur, US et ICH , ainsi que d’une expertise dans la constitution de dossiers d’AMM. Une connaissance des petites molécules, toutes voies d’administration, est vivement souhaitée.

Vous savez être rigoureux, autonome et faites preuve d’esprit de synthèse. Vous aimez effectuer des tâches multiples dans un environnement évolutif et créatif.

Au-delà des strictes compétences professionnelles, ORPHELIA Pharma recherche une personne enthousiaste, stratège, entreprenante, dotée de leadership, combinant aisance, rigueur, qualités humaines et bonnes aptitudes de communication.

Une excellente maîtrise de l’anglais scientifique et technique est indispensable pour la bonne tenue du poste.

Les missions qui vous seront confiées seront les suivantes :

  • Définition de la stratégie réglementaire en collaboration avec la Direction,
  • Interface avec les autorités de santé et négociation pour l’enregistrement et la vie des produits,
  • Constitution des dossiers de demande d’AMM et suivi des procédures d’ enregistrement (eCTD) auprès des autorités de santé internationales jusqu’à l’obtention de l’AMM,
  • Suivi de la vie des produits en post-AMM (variations, renouvellements, etc.),
  • Information Produit et articles de conditionnement : création, mise à jour et validation,
  • Constitution des dossiers de transparence et de prix,
  • Participation aux inspections par des autorités de santé,
  • Veille réglementaire,
  • Conseil et assistance sur les aspects réglementaires auprès des services concernés (qualité, marketing, ventes, logistique etc.),
  • Participation à la mise en place et au suivi d’études cliniques : rédaction des dossiers de demande d’autorisation ; validation de documents techniques en relation avec la R&D (IMPD, IB, protocoles cliniques).

En outre, vous devrez être capable de bien gérer l’interface avec les équipes internes et les sous-traitants.

Poste basé à Paris.

H/F

Rémunération en fonction de l’expérience, Bonus, BSPCE

Vous pouvez adresser votre candidature à : contact@orphelia-pharma.eu

Kigabeq® launch in Germany

ORPHELIA Pharma and DESITIN GmbH announce the launch of Kigabeq® in Germany

Paris/Lyon and Hamburg, July 16, 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 the epilepsy specialist Desitin GmbH to market Kigabeq® in Germany.

Kigabeq® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug, available as scored soluble tablets of 100 mg and 500 mg strengths for oral and (naso)gastric administration. Kigabeq® has been developed exclusively for children and has been granted a Paediatric Use Marketing Authorization (PUMA) by the European Commission in October 2018. Kigabeq® is the first-line treatment for infantile spasms, an extremely severe early childhood encephalopathic epilepsy.

« This is a key milestone for ORPHELIA Pharma and 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 we are convinced that it will improve the life of children affected by this condition » comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma.

« Kigabeq® is an appropriate therapeutic option and is highly appreciated as new part of our antiepileptic portfolio », Martin Zentgraf, General Manager of Desitin says. « We have started promotion of Kigabeq® in July 2019 already which will hopefully be well accepted for its use in pediatric patients ».

« As a renowned specialist in epilepsy, Desitin is the best possible partner for Kigabeq® in Germany » adds Gilles Alberici, President of ORPHELIA Pharma. « Germany is the first country where we launch our first approved product. We are now preparing the market access in France and ORPHELIA Pharma looks forward to building relationships with other distribution partners to market Kigabeq® throughout Europe. This agreement marks our transition from a development to a commercial-stage company ».

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

Based in Paris and Lyon, ORPHELIA Pharma is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament. For more information, please visit www.orphelia-pharma.eu

About Desitin GmbH – CNS in Focus; People in Mind

Based in Hamburg, Desitin Arzneimittel GmbH is marketing pharmaceutical products in international countries. At our Hamburg plant we produce sophisticated products for the treatment of neurological and psychiatric illnesses and offer a large number of established and innovative products, especially for our core indications epilepsy and Parkinson’s syndrome. The optimisation of pharmacotherapy through continuous development and in cooperation with other future-oriented companies, is one of Desitin’s main areas of activity and investment. For more information, please visit https://www.desitinpharma.com

Gustave Roussy Cancer Centre and Orphelia Pharma

ORPHELIA Pharma executes a collaboration and license agreement with Gustave Roussy cancer centre for the development of the first pediatric formulation of temozolomide

Paris and Lyon, July 2nd, 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 a collaboration and license agreement has been executed with Gustave Roussy, the leading European Comprehensive Cancer Centre for the development of Kimozo®, the first paediatric formulation of temozolomide.

Temozolomide is an essential anticancer medicine authorized for the treatment of malignant glioma in children. Unfortunately, no paediatric formulation exists; hence caregivers are asked to open the temozolomide adult capsules and mix the medicine in soft food. The need for a paediatric formulation has been expressed by the European Medicine Agency in their document « Draft Inventory of paediatric therapeutic needs – EMA/381728/2014 ».

« Temozolomide is one of the anticancer medicines for which a real unmet medical need for a paediatric formulation exists », explained Pr. Gilles Vassal, Director of Clinical Research at Gustave Roussy. « This is widely recognized by the international paediatric oncology community and the reason why Gustave Roussy has developed an innovative hospital compounded preparation for children. »

Kimozo® was developed starting from Gustave Roussy hospital-compounded preparation and is the first paediatric formulation of temozolomide to be presented as a taste-masked oral suspension. A sealed bottle and five oral syringes will allow an efficient and safe dispensing to children. ORPHELIA Pharma is currently monitoring industrial development of Kimozo®.

« When using the adult formulation of temozolomide, the drug could be regurgitated by the child and the administered dose is not precise. Furthermore, during the preparation of the drug, caregivers and patient’s family could be exposed to this cytotoxic molecule. Gustave Roussy has developed a very useful hospital-compounded preparation », comments Jérémy Bastid, Chief Development Officer at ORPHELIA Pharma.

« The medical need for temozolomide is very significant in children », highlights Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « Beside malignant glioma, this medicine is used in several rare childhood cancers. Thus, Kimozo® will be helpful in multiple indications.»

About ORPHELIA Pharma

Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament. For more information: www.orphelia-pharma.eu

About Gustave Roussy

Gustave Roussy, the leading cancer centre in Europe, is a comprehensive hub of expertise in oncology, entirely devoted to patients. It employs 3,100 professional staff engaged in patient care, research and teaching. For more information : https://www.gustaveroussy.fr/en