The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) for the treatment of specific rare diseases. Bkemv is approved for the same indications as Soliris, including the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Addressing Rare Diseases

Sarah Yim, director of the Office of Therapeutic Biologics and Biosimilars at the FDA’s Center for Drug Evaluation and Research, emphasized the importance of developing interchangeable biosimilar treatments for rare, life-threatening conditions. She stated, “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.”

PNH and aHUS are rare diseases affecting fewer than 200,000 people in the U.S. Both conditions involve the breakdown of red blood cells, leading to severe complications such as anemia, thrombosis, pancytopenia, and kidney failure.

Mechanism and Safety

Bkemv is a monoclonal antibody that binds to the complement C5 protein, inhibiting the activation of the complement system and preventing intravascular hemolysis in patients with PNH and aHUS. Similar to Soliris, Bkemv carries a Boxed Warning regarding the increased risk of serious meningococcal infections. Patients should complete meningococcal vaccination before starting Bkemv or Soliris and be monitored for signs of infection.

Adverse Reactions and REMS Program

Bkemv shares the same safety profile as Soliris, with adverse reactions in PNH patients including headache, nasopharyngitis, back pain, and nausea. In aHUS patients, common adverse reactions include headache, diarrhea, hypertension, and upper respiratory infections. Bkemv is available only through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS) to ensure the benefits outweigh the risks.

Biosimilar Approval and Market Impact

As the 53rd approved biosimilar in the U.S., and the 13th interchangeable biosimilar, Bkemv meets the FDA’s stringent approval standards, ensuring no clinically meaningful differences from Soliris. This approval supports the FDA’s commitment to a competitive marketplace, offering patients more affordable treatment options. Bkemv’s approval was granted to Amgen Inc.

Highlights

• FDA Approval: Bkemv approved as the first interchangeable biosimilar to Soliris.
• Indications: Treats paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
• Mechanism: Monoclonal antibody inhibiting complement C5 protein to prevent red blood cell breakdown.
• Safety: Boxed Warning for serious meningococcal infections; available through a restricted REMS program.
• Market Impact: Supports competitive marketplace and patient access to affordable treatments.
• Approval Granted to: Amgen Inc.