FDA and EMA accept filings for higher dose of nusinersen in SMA

23 January 2025

The US Food and Drug Administration (FDA) has accepted Biogen’s (Nasdaq: BIIB) supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen, marketed as Spinraza, for spinal muscular atrophy (SMA).

The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50mg doses 14 days apart, and higher maintenance regimen, 28mg, every four months, compared to the approved nusinersen regimen.

“We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, head of the Neuromuscular Development Unit at Biogen. “This milestone reflects our steadfast commitment to advance treatment options for individuals with SMA, and we expect that this higher dose regimen will offer meaningful benefits to patients and their families. We are deeply thankful for the unwavering support of the trial participants, their families, site staff, and the SMA community without whom these advancements would not have been possible,” she added.

This article is accessible to registered users, to continue reading please register for free.  A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Login to your account

Become a subscriber

 

£820

Or £77 per month

Subscribe Now
  • Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
  • Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
  • Daily roundup of key events in pharma and biotech.
  • Monthly in-depth briefings on Boardroom appointments and M&A news.
  • Choose from a cost-effective annual package or a flexible monthly subscription
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK

Companies featured in this story

More ones to watch >


Today's issue

Company Spotlight





More Features in Biotechnology