OCTALFA announces the inclusion of the first patient in the French Early Access Program granted to its subsidiary ORPHELIA Pharma for the use of KIMOZO® 40 mg/ml (temozolomide oral suspension) in the treatment of relapsed or refractory neuroblastoma

On June 22, 2022 OCTALFA reported 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 (Press release, ORPHELIA Pharma, JUN 22, 2022, View Source;utm_medium=rss&utm_campaign=octalfa-announces-the-inclusion-of-the-first-patient-in-the-french-early-access-program-granted-to-its-subsidiary-orphelia-pharma-for-the-use-of-kimozo-40-mg-ml-temozolomide-oral-suspension-in [SID1234616508]).

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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.