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

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.