FDA Approval Gives Third Option to Treat Rare, Deadly Disease

The FDA recently approved a third agent to treat paroxysmal nocturnal hemoglobinuria (PNH). With some conditions, that number of treatments may prompt payer preferencing, but that is unlikely to happen with this ultra-rare, potentially fatal disease, observe industry experts.

On May 14, the FDA approved Apellis Pharmaceuticals, Inc.’s Empaveli (pegcetacoplan) as the first and only targeted C3 therapy for the treatment of adults with PNH. It is approved for both treatment-naïve people, as well as ones switching from any C5 inhibitor. The FDA has approved two of those drugs: Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz), both from Alexion Pharmaceuticals, Inc., which was just acquired by AstraZeneca in July.

PNH is an acquired, life-threatening blood disorder that causes the destruction of red blood cells, formation of blood clots and impairment of bone marrow function. It is an ultra-rare disease, impacting about 1 to 1.5 people per million, according to Johns Hopkins’ Sidney Kimmel Comprehensive Cancer Center. The median age of diagnosis is between 35 and 40 years old, with a 10-year median survival after diagnosis, although some people may live for decades with minor symptoms. Blood tests that can confirm the diagnosis are available.

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