BioPharma, Pharma

FDA accepts Alexion’s application for next-generation PNH drug

Soliris, the drugmaker's cash cow approved for the same disease, faces biosimilar competition as patent expiry looms.

The Food and Drug Administration has accepted for priority review Alexion Pharmaceuticals’ new drug for a rare blood disorder.

The New Haven, Connecticut-based company said Monday that the FDA would review its application for ALXN1210, a C5 complement inhibitor in Phase III development for paroxysmal nocturnal hemoglobinuria, or PNH. The disease causes chronic, uncontrolled activation of part of the immune system known as the complement system, resulting in destruction of red blood cells and anemia, fatigue and other symptoms, including internal blood clots that can cause organ damage and death. PNH occurs in between 0.5-1.5 per 1 million people in the general population, according to the National Organization for Rare Disorders.

ALXN1210 is a long-acting medicine with the same target as Soliris (eculizumab), for which Alexion won FDA approval for reducing red blood cell destruction – also known as hemolysis – in PNH in 2007, but which must be dosed more frequently. Soliris is also approved for atypical hemolytic uremic syndrome, or aHUS, and anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (anti-AchR+ gMG).

When it was approved, Soliris cost more than $400,000 and held the title of most expensive drug in the world. Since then, its price has risen to more than $500,000, according to news reports, though other drugs have since hit the market with higher list prices. Now, however, Soliris faces the prospect of patent expiration and biosimilar competition.

According to Generics and Biosimilars Initiative, a group formed 10 years ago by Pro Pharma Communications International, Soliris’ US patents will expire in March 2021. In Europe, where the patents will expire in May 2020, Amgen is running a Phase III study of its biosimilar version of Soliris, ABM 959, enrolling PNH patients in several European Union countries, along with Russia, Japan and Korea, according to the European Clinical Trials Registry. Meanwhile, the Australian New Zealand Clinical Trials Registry lists a Phase III pharmacokinetics study comparing ABP 959 with Soliris among healthy male subjects in Australia.

Alexion noted in its latest quarterly earnings filing that it relies on Soliris for a substantial portion of its revenue, but expects increased competition from biosimilars, hence its efforts to build out its pipeline. Soliris’ sales in 2017 were $3.14 billion, compared with $2.84 billion in 2016, according to the company’s annual report. Its other marketed products are Strensiq (asfotase alfa), which had 2017 sales of $339.8 million, and Kanuma (sebelipase alfa), whose sales were $65.6 million; both drugs treat rare metabolic diseases.

According to the company’s pipeline page, ALXN1210 is the only premarket pipeline drug currently in clinical trials, while two others are in preclinical development. Earlier this year, topline Phase III results in treatment-naive patients showed the drug was successful in achieving non-inferiority to Soliris on the co-primary endpoints of transfusion avoidance and lactate dehydrogenase normalization, while topline results from another Phase III study showed PNH patients could safely and effectively switch from Soliris to ALXN1210. The company also has ALXN1210 in clinical trials for aHUS and gMG.

Photo: wikimedia commons

Shares1
Shares1