Alexion's Soliris gets US approval for myasthenia gravis

Alexion has finally claimed FDA approval for its complement inhibitor Soliris in myasthenia gravis (MG), potentially adding another $1bn-plus in peak sales for the product.

The US regulator has approved Soliris (eculizumab) to treat adults with refractory generalised MG who are anti-acetylcholine receptor (AchR) antibody-positive, a patient group that “continues to suffer from significant unresolved disease symptoms despite existing treatment options”, according to Alexion’s head of R&D John Orloff.

Soliris was tested in clinical trials in patients with the neuromuscular disease who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms such as difficulties seeing, walking, talking, swallowing and breathing. Refractory AChR-positive patients represent approximately 5% to 10% of the total MG population of 60,000 to 80,000 people in the US.

The new indication represents the first foray for Alexion in neurology and a massive development for MG patients in the US, who have not seen a therapy approved for 60 years, according to Muscular Dystrophy Association (MDA). The European Commission approved use of the drug in MG in August and it was swiftly launched in Germany.

Chief executive Lynn O’Connor Vos, described the last few years as an “unprecedented time” in neuromuscular disease research, with “six FDA approvals for drugs to treat neuromuscular diseases in MDA’s programme - including periodic paralysis, Duchenne muscular dystrophy, amyotrophic lateral sclerosis, spinal muscular atrophy and now MG”.

Leerink analysts reckon the new indication - which comes after approvals for paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome - could contribute $1.2bn out of a predicted $5.3bn in sales for Soliris within five years.

The product is already a big earner for Alexion, which estimates sales will top $3bn this year, although some analysts have suggested that its high price - which could reach the $700,000 mark for MG patients depending on the dose required - could lead to resistance from payers.

The company has already taken measures to try to reduce its reliance on the 10-year-old cash cow product however, launching a restructuring drive to cut costs and increase the flow of new products - and licensing deals - from its pipeline.

The revamp was announced after Soliris failed to hit the mark in a trial in kidney transplant complications and allegations of sales fraud, which was followed by the departures of CEO David Hallal - to be replaced by former AstraZeneca CEO David Brennan - and CFO Vikas Sinha.