Alexion snags EU yes for new Soliris indication
- Alexion Pharmaceuticals Inc. has secured EU approval for Soliris in the treatment of a very small subgroup of patients with a rare, muscle-weakening disease.
- Soliris is now the first complement-based therapy green lighted in Europe for adults with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, according to an Aug. 21 statement from Alexion.
- The approval hinged on clinical data from the Phase 3 REGAIN investigation and its open-label extension study. Alexion plans to launch Soliris for this latest indication first in Germany and later in other European countries. A Supplemental Biologics License Applications (sBLA) for Soliris in the gMG setting is currently under review in the U.S.
Following the earlier recommendation from the European Medicines Agency, the approval from the European Commission is a piece of much-needed good news for Alexion, which is trying to turn the page on problems related to the marketing of Soliris.
Despite the ongoing challenges, sales have picked up. Alexion reported better-than-expected second quarter 2017 results, with the bulk of its income coming from $814 million worth of Soliris (eculizumab) sales, a 16% increase year over year. Overall sales for 2017 may reach as much as $3.125 billion, according to the company. Alexion has also beefed up its star performer's patent portfolio, with three new patents disclosed earlier this month.
Myasthenia gravis is an autoimmune disease that affects around 20 in 100,000 people in the U.S., though it is likely underdiagnosed. The disease often progresses to a generalized form, gMG. Around 10-15% of patients don't respond to treatment, causing them to develop severe and potentially life-threatening symptoms, such as respiratory muscles weakening so much that a patient can't adequately breathe.
There are a handful of other drugs in development for myasthenia gravis. Belgian biopharma UCB is carrying out a Phase 2a trial with rozanolixizumab, with first results expected in the second quarter of 2018.
Georgetown Translational Pharmaceuticals — currently in the process of being acquired by Oxis International Inc. — is developing GTP-004, a fixed-dose combination of the approved product pyridostigmine (used for MG patients) with an undisclosed gastrointestinal drug to reduce the side effects of pyridostigmine. A Phase 1 proof-of-concept study for the combination is planned for late 2017 or early 2018.
- Alexion Pharmaceuticals Press release
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