May 14, 2021

The U.S. FDA has approved Empaveli™ (pegcetacoplan), manufactured by Apellis Pharmaceuticals, to treat adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH).

PHN is a rare, potentially fatal blood disorder. A genetic mutation causes destruction of red blood cells and contributes to anemia, blood clots, and impaired bone marrow function. Median survival following diagnosis is 10 years, though individuals can live longer. Most individuals with PNH receive a diagnosis at 35-40 years of age. Treatment options include blood transfusions to replace destroyed red blood cells.

In a clinical study, 85% of patients who received Empaveli were transfusion free at Week 16 compared to 15% on Soliris® (eculizumab – Alexion), a current standard of care. Empaveli also delivered a superior change from baseline in hemoglobin levels compared to Soliris and demonstrated non-inferiority to Soliris in transfusion avoidance.

Recommended dosing is 1,080mg given via subcutaneous (SC) infusion twice a week using a commercially available pump with a 20mL reservoir. Empaveli can be administered at home by the patient or a caregiver once the individual has been trained in proper SC infusion technique by a healthcare provider.

A black box warning cautions that meningococcal infections may occur in patients treated with Empaveli and these infections may quickly become life-threatening or fatal if not recognized and treated early. Empaveli may predispose individuals to serious infections, particularly those caused by encapsulated bacteria such as Streptococcus pneumoniae; Neisseria meningitidis types A, C, W, Y, and B; and Haemophilus influenzae type B.

Apellis has launched Empaveli at a wholesale acquisition cost (WAC) of $458,000 per year.

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