Crigler-Najjar Syndrome: Genethon obtains PRIME priority drug status from the EMA for its gene therapy

The European Medicines Agency (EMA) has granted PRIME (PRIority MEdicine) status to gene therapy product GNT-0003 currently being tested as part of a clinical trial in Crigler-Najjar syndrome, a rare liver disease.

PRIME (PRIority MEdicines) status is granted to drug candidates with major therapeutic potential. It follows the conclusive results of the first phases of the trial conducted by Généthon in three countries in Europe (France, Italy, Netherlands).
With this status, the developer of the drug candidate benefits from early and proactive support from the EMA throughout the clinical development, support which makes it possible to accelerate the evaluation of the latter to put it at the earliest disposal of patients.

Read the press release