(Reuters) – A committee of the European health regulator on Friday recommended approving Swiss drugmaker Novartis AG’s gene therapy Zolgensma for a muscle wasting disease in babies and young children.
The human medicines committee (CHMP) of the European Medicines Agency have recommended https://bit.ly/3ajanId conditional marketing authorization for the therapy, used for treating a rare and often fatal genetic disease called spinal muscular atrophy (SMA).
Novartis has been under pressure since the treatment, the world’s most expensive at $2.1 million, came under scrutiny in August over a data manipulation scandal that prompted a U.S. Food and Drug Administration (FDA) investigation.
While final approvals are up to the European Commission, it generally takes and endorses CHMP’s recommendation within a couple of months.
Zolgensma is already approved in the United States for children aged up to two with SMA, and is infused into patients’ veins.
(Reporting by Pushkala Aripaka and Tanishaa Nadkar in Bengaluru; Editing by Shinjini Ganguli)