FDA Approves First Interchangeable Biosimilar for Rare Diseases
May 28, 2024
The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab). This approval is significant for treating rare diseases, particularly paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
Key Points:
- Treatment Indications:
- PNH: Reduces hemolysis (breakdown of red blood cells).
- aHUS: Inhibits complement-mediated thrombotic microangiopathy.
- About the Conditions:
- PNH: Causes anemia, blood clots, pancytopenia, and dark urine.
- aHUS: Leads to anemia, low platelet count, and kidney failure.
- Mechanism:
- Bkemv is a monoclonal antibody that binds to the complement C5 protein, preventing red blood cell breakdown in PNH and aHUS patients.
- Safety Concerns:
- Both Bkemv and Soliris carry a Boxed Warning for increased risk of serious meningococcal infections. Patients must complete meningococcal vaccination before starting treatment and be monitored for infection symptoms.
- Side Effects:
- PNH Trials: Headache, nasopharyngitis, back pain, nausea.
- aHUS Trials: Headache, diarrhea, hypertension, upper respiratory infections, among others.
- Approval and Access:
- Bkemv is available through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS).
- Regulatory Milestone:
- Bkemv is the 53rd approved biosimilar in the U.S. and the 13th interchangeable biosimilar. This approval allows for “pharmacy-level substitution” without consulting the prescriber, subject to state laws.
Impact: This approval highlights the FDA’s commitment to facilitating the development of biosimilars, which increases access to affordable treatment options for patients with rare diseases. The FDA ensures that both biosimilars and interchangeable biosimilars meet rigorous safety and effectiveness standards.
The approval of Bkemv is a significant step towards a more competitive marketplace for biological products, potentially lowering costs and expanding treatment access for patients with limited options.
Approved by: The FDA granted the approval of Bkemv to Amgen Inc