FDA OKs first-of-its-kind treatment for paroxysmal nocturnal hemoglobinuria

Christine Blank

OptumRx had identified pegcetacoplan as one of the top 5 drugs in the pipeline this year.

FDA approved pegcetacoplan (Empaveli, Apellis), the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

PBM OptumRx had identified the medication as one of the top 5 drugs that will have an impact this year in its Drug Pipeline Insights Report, saying it could become the market leader in PNH treatments.

Related: Top 5 drugs in the pipeline that could have an impact this year

PNH is a rare, chronic, life-threatening blood disorder caused by an acquired mutation, which leads to uncontrolled complement activation and the destruction of red blood cells through intravascular and extravascular hemolysis.

Empaveli is approved for adults with PNH who are treatment naïve as well as patients switching from the C5 inhibitors eculizumab (Soliris, Alexion Pharmaceuticals) and ravulizumab (Ultomiris, Alexion).

“As the first, FDA-approved targeted C3 therapy, Empaveli has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naïve patients,” said Cedric Francois, M.D., Ph.D., co-founder and CEO of Apellis, in a press release. “This approval represents a major scientific advancement as Empaveli ushers in the first new class of complement medicine in almost 15 years.”

Related: Novel pair of therapies offers hope to patients with rare blood disorders

Empaveli has the potential to improve the lives of patients with PNH by increasing hemoglobin and reducing blood transfusion requirements, added Olga Frankfurt, M.D., PEGASUS study investigator and associate professor in the department of medicine, division of hematology and oncology at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

In the head-to-head Phase 3 PEGASUS study, Empaveli met the primary endpoint, demonstrating superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin. Notably, 85% of Empaveli-treated patients were didn't need transfusions over 16 weeks versus 15% of Soliris-treated patients

The prescribing information for Empaveli contains a boxed warning that the drug may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. FDA has approved a risk evaluation and mitigation strategy (REMS) for the treatment.

Read more: FDA clears treatment for rare obesity disorders