Pharma Focus Asia

FDA Approves Initial Interchangeable Biosimilar for Two Rare Conditions

Wednesday, May 29, 2024

Today, the U.S. Food and Drug Administration (FDA) authorized Bkemv (eculizumab-aeeb) as the inaugural interchangeable biosimilar to Soliris (eculizumab) for treating specific rare diseases. Bkemv has been endorsed for treating the following indications, which are presently also approved for Soliris:

  1. Treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.
  2. Treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Sarah Yim, director of the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research, stated, "Many rare conditions are life-threatening, and many do not have treatments." She emphasized the FDA's commitment to facilitating the development of safe and effective interchangeable biosimilar treatments to expand access for individuals with rare diseases.

PNH and aHUS are considered rare diseases if they affect fewer than 200,000 people in the U.S. Both conditions involve the breakdown of red blood cells, with PNH resulting in anemia, thrombosis, pancytopenia, and dark urine, while aHUS leads to anemia, thrombocytopenia, and kidney failure.

Bkemv functions as a monoclonal antibody binding to the complement C5 protein, inhibiting the activation of the complement system, an integral part of the body’s immune system. This mechanism prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

Similar to Soliris, Bkemv carries a Boxed Warning highlighting the increased risk of serious and life-threatening meningococcal infections. Patients are advised to complete meningococcal vaccination before starting either medication, be monitored for early signs of meningococcal infections, and undergo immediate evaluation if signs of infection arise.

Bkemv, being an interchangeable biosimilar, exhibits high similarity with no clinically meaningful differences compared to Soliris. It shares the same safety warnings and is expected to evoke similar adverse reactions. Adverse reactions reported in clinical trials for Soliris include headache, nasopharyngitis, back pain, nausea, diarrhea, hypertension, and others.

The distribution of Bkemv is limited to a restricted program known as the Bkemv Risk Evaluation and Mitigation Strategy (REMS), aimed at ensuring the benefits of the medication outweigh its risks.

This approval marks the 53rd biosimilar to be approved in the U.S., with 13 of them being approved as interchangeable biosimilars. Interchangeable biosimilars, meeting stringent requirements, can be substituted for the reference product without consulting the prescriber, similar to generic drug substitution for brand name drugs.

Overall, the approval of biosimilar and interchangeable biosimilar products aligns with the FDA's commitment to fostering a competitive marketplace for biological products and enhancing patient access to more affordable treatment options.

The approval of Bkemv was granted to Amgen Inc.



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