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.