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OCTALFA announces inclusion of the first patient in the French EAP of KIMOZO

6 July 2022

Lyon, June 22nd, 2022 – OCTALFA announces that its subsidiary ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of orphan medicines for the treatment of rare and pediatric diseases, has included the first patient in the KIMOZO® French Early Access Program for the treatment of relapsed or refractory neuroblastoma.

KIMOZO® 40 mg/ml (oral suspension of temozolomide) was granted an early access pre-marketing authorization (Autorisation d’Accès Précoce, AAP) in France on March 31st, 2022 by the Haute Autorité de Santé (HAS) for the treatment of relapsed or refractory neuroblastoma in patients aged 1 to 6 years as well as patients unable to swallow temozolomide capsules.

The goal of Early Access Programs in France is to accelerate access to innovative drugs before or after Market Authorization is granted once all conditions specified in Article L.5121-12 of the French Public Health Code (Code de la Santé Publique) are met:

  • No appropriate treatment exists;
  • The treatment’s implementation cannot be postponed;
  • The efficacy and safety of these drugs are strongly presumed based on therapeutic trials;
  • These drugs are considered to be innovative, particularly when compared to a possible clinically-relevant comparator.

Full details of the early access program can be found at the Haute Autorité de Santé and the ANSM websites.

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed to treat rare pediatric cancers.

In France, KIMOZO® has been granted an Early Access Program, to be used as monotherapy or in combination with irinotecan or topotecan in the treatment of pediatric patients aged 1 to 6 years and in patients with incapacity to swallow capsules of temozolomide and who suffer from:

  • refractory high-risk neuroblastoma or presenting an insufficient response to induction chemotherapy;
  • relapsed high-risk neuroblastoma after at least a partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.

KIMOZO® was developed by ORPHELIA Pharma in collaboration with Gustave Roussy.

About ORPHELIA Pharma

Founded by Hugues Bienaymé, ORPHELIA Pharma is a biopharmaceutical company based out of Paris and Lyon which develops and markets drugs for the treatment of pediatric and rare diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the rare diseases therapeutic area, in the form of formulations adapted to a pediatric population. With two drugs approved in the European Union, and another investigational medicine in a late stage of clinical development, ORPHELIA Pharma has recently established regional agreements in Europe, the United States and China, and is conducting research projects through academic and industrial partnerships.

For more information, please visit


Set up by Gilles Alberici, OCTALFA is an independent family-owned company that has been developing and innovating for 15 years in the fields of life sciences, biotechnology and healthcare. Thanks to our subsidiaries and participations, we assert our presence in branches of medicines, particularly in pediatrics, cancer immunology, diagnostics and nanomaterials. We also provide support to patients living with cancer and people with disabilities, particularly children, in France and Madagascar, thanks to our endowment fund and the Dominique & Tom Alberici – OCTALFA Corporate Foundation, whose actions have been fully integrated into OCTALFA’s governance strategy since 2008.

OCTALFA is a shareholder of ORPHELIA Pharma since 2015.

For more information, please visit


Charlotte Maddalena / e-mail: / phone: +33 (0)4 37 49 87 20