Novartis: EC approval for Zolgensma

(CercleFinance.com) - AveXis, a Novartis company, has received EC approval and is activating the "Day One" access program for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA).


Zolgensma is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and for the clinical diagnosis of Type 1 SMA.

AveXis is in discussions with EU governments and reimbursement agencies to agree the terms of the innovative "Day One" access program to allow rapid access in all EU countries given the urgent need for treatment.

In Europe, each year about 550-600 infants are born with this disease, a rare genetic neuromuscular disease caused by the absence of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions including breathing and swallowing.


Copyright (c) 2020 CercleFinance.com. All rights reserved.