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)

6 January 2020

ORPHELIA Pharma will be present at Toulouse for the thirtieth SFNP congress
6 January 2020

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)
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10 December 2019

Orphan Drug Designation granted by FDA for temozolomide in the treatment of neuroblastoma
10 December 2019

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 […]
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26 November 2019

Granting of the Marketing Authorisation for Ivozall
26 November 2019

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, […]
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29 October 2019

ORPHELIA Pharma will be present at CPhI in Frankfurt
29 October 2019

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).
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