FDA Approves Bkemv, A Potentially Game-Changing Treatment for Rare Blood Diseases

U.S. Food and Drug Administration

WASHINGTON, D.C. — In a significant advancement for patients with rare blood diseases, the U.S. Food and Drug Administration (FDA) has given its nod to Bkemv (eculizumab-aeeb), the first interchangeable biosimilar to Soliris (eculizumab), on May 28, 2024. These diseases, named paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), are the latest rare diseases to gain an innovative, FDA-approved treatment.

PNH and aHUS afflict fewer than 200,000 people in the U.S., classifying them as rare diseases. They lead to the destruction of red blood cells, initiating a cascade of distressing symptoms like low blood cell counts, blood clot formations, dark urine, and kidney failure. “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.

Bkemv, a monoclonal antibody, interferes with the triggering of the complement system, a segment of the body’s immune response. By attaching itself to the C5 protein and inhibiting the system, Bkemv prevents the undue disintegration of red blood cells in people with PNH and aHUS.

However, the newly approved medical product comes with stringent safety precautions. It heightens the risk of severe and potentially lethal infections, including those caused by Neisseria meningitidis, the bacterium behind meningitis. To combat this, patients are advised to complete meningococcal vaccinations before initiating treatment with Bkemv or Soliris. Doctors must also vigilantly monitor these patients for early signs of infection.

The introduction of Bkemv in the healthcare market is significant as it represents a biosimilar that’s not only effective but interchangeable, indicating it mirrors Soliris in function without any clinically consequential disparities. This likeness spans safety warnings, expected side effects, and overall efficacy. Commonly reported side effects include headaches, back pain, the common cold, and nausea.

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Bkemv’s distribution will observe strict protocols under the Bkemv Risk Evaluation and Mitigation Strategy (REMS), a safety program for drugs with serious safety concerns. It ensures the medication’s benefits exceed the potential risks.

Bkemv’s approval heralds the 53rd biosimilar greenlit in the U.S., with 13 of these interchangeable. An interchangeable biosimilar can replace the reference product without needing the physician’s consultation. This substitutability can occur at the pharmacy level, mirroring the substitution of generic drugs for brand-name counterparts.

Behind this landmark approval is the FDA’s enduring commitment to fostering a competitive environment for biological products and amplifying patients’ access to affordable treatments. It demonstrates the rigorous nature of the FDA approval process, reassuring healthcare providers that both biosimilar and interchangeable biosimilar products are safe and effective.

The credit for bringing Bkemv to patients goes to Amgen Inc., marking a significant stride in the wider attempt to provide effective treatment options for patients with rare, life-threatening diseases.

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