FDA Approves First Interchangeable Biosimilar to Eculizumab for PNH and aHUS

The US Food and Drug Administration has announced the approvals of eculizumab-aeeb (Bkemv) as an interchangeable biosimilar to eculizumab (Soliris) for the treatment of treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis as well as atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Announced on May 28, 2024, the decision marks the agency’s 53rd approval for a biosimilar in the US but the first interchangeable biosimilar approval to eculizumab in the treatment of PNH to reduce hemolysis and aHUS for inhibiting complement-mediated thrombotic microangiopathy.

“Many rare conditions are life-threatening, and many do not have treatments,” said Sarah Yim, director of the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research. “The FDA is committed to help facilitate the development of safe and effective interchangeable biosimilar treatments that can expand access for individuals with rare diseases whose current treatment options are limited.”

A complement inhibitor from Alexion Pharmaceuticals, eculizumab (Soliris) boasts approvals from the FDA dating back nearly 2 decades, with its first indication for PNH coming in March 2007. In the next 17 years, the agent would receive approval for all patients with aHUS, generalized myasthenia graves, and neuromyelitis optic spectrum disorder in 2011, 2017, and 2019, respectively.

According to the FDA, eculizumab-aeeb will have the same Boxed Warning as its reference agent, which denotes its associated with serious and life-threatening meningococcal infections caused by Neisseria meningitidis. The FDA notes patients who have have completed meningococcal vaccination before startingeculizumab-aeebor eculizumab should be monitored for early signs and symptoms of meningococcal infections and undergo further evaluation immediately if signs of infection develop.

The FDA also pointed out eculizumab-aeeb will only be available through a Risk Evaluation and Mitigation Strategy (REMS) program. The approval of eculizumab-aeeb as the first interchangeable biosimilar to eculizumab was awarded to Amgen Inc.

References:

1. U.S. Food and Drug Administration. FDA approves first interchangeable biosimilar for two rare diseases [news release]. FDA website. https://www.fda.gov/news-events/press-announcements/fda-approves-first-interchangeable-biosimilar-two-rare-diseases. Published May 28, 2024. Accessed May 28, 2024.
2. Stewart J. History of Soliris. Drugs.com website. https://www.drugs.com/history/soliris.html. Published September 7, 2020. Accessed May 28, 2024.