FDA lets Soliris through in new indication but take-up not guaranteed

25 October 2017
2019_biotech_test_vial_discovery_big

The US Food and Drug Administration’s (FDA) approval of Soliris (eculizumab) in a new indication on Monday was billed as an exciting step forward for a long underserved patient group, but lukewarm investor reaction since reflects a market unsure of whether broad uptake will follow.

Soliris, which was developed by US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN), is already approved in the USA for two rare blood disorders. This time it received the nod in generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AchR) antibody-positive, making it the first FDA-approved treatment for the chronic and debilitating neuromuscular disorder in more than 60 years.

The approval was based on the company's Phase III REGAIN trial in which Soliris missed its primary endpoint, but it did achieve statistical significance and multiple secondary endpoints were successful. The drug showed treatment benefits for patients with anti-AchR antibody-positive gMG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms, which can include difficulties seeing, walking, talking, swallowing and breathing.

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



Today's issue

Company Spotlight





More Features in Biotechnology