The French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d’Utilisation de cohorte (cohort ATU), or Temporary Authorization for Use, for the use of berotralstat to prevent attacks of HAE in appropriate patients aged 12 and older.
This cohort ATU allows patients with HAE in France to receive treatment with berotralstat before the drug is granted marketing authorization by the European Commission (EC).
According to the French Code of Public Health, a cohort ATU is dedicated to high unmet medical needs in serious diseases and covers medicinal products of which the efficacy and safety of use are strongly presumed and intended for a group or sub-group of patients treated and monitored in accordance with criteria defined in a protocol for therapeutic use and collection of information.
“HAE is a debilitating and potentially deadly disease so the early access to a new treatment option for patients is exciting news,” said Dr. Laurence Bouillet, head of internal medicine, University Hospital Grenoble, Coordinating Reference Centre for HAE in France (CREAK).
“The cohort ATU represents BioCryst’s second early access program approved in Europe and provides patients in France with faster access to treatment,” says Jon Stonehouse, CEO of BioCryst Pharmaceuticals, Inc.
The EC will review the CHMP recommendation and a final approval decision from the EC on the marketing authorization application (MAA) for Orladeyo is expected in the second quarter.(Source: BioCryst)