Saturday, June 22, 2024

FDA Approves Bkemv: Cost-Saving Biosimilar for Rare Diseases

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The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) for treating certain rare diseases. Cost-saving biosimilars like Bkemv play a crucial role in managing healthcare expenditures by providing more affordable treatment options. These alternatives not only help reduce the financial burden on the healthcare system but also improve access to essential medications for patients.

Bkemv is now approved for treating paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, which is the destruction of red blood cells. PNH is a rare and potentially life-threatening blood disorder characterized by severe anemia, thrombosis (blood clots), and other complications. By reducing hemolysis, Bkemv helps alleviate the symptoms and improve the quality of life for patients with PNH.

In addition, Bkemv is approved for treating atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, a condition where small blood clots form in the blood vessels, leading to kidney failure and other severe complications. aHUS is another rare and life-threatening disease, and effective treatment options are limited. Bkemv’s approval provides a critical new option for managing this condition, potentially preventing serious health outcomes and improving patient survival rates.

The approval of Bkemv as an interchangeable biosimilar means that it can be substituted for Soliris at the pharmacy level without consulting the prescriber, subject to state pharmacy laws. This interchangeability is significant because it can increase the availability and use of Bkemv, leading to further reductions in healthcare expenditures. The introduction of interchangeable biosimilars like Bkemv supports the FDA’s goal of fostering a competitive marketplace for biological products, ultimately benefiting patients through increased access to safe, effective, and more affordable treatments.

Impact on Rare Diseases

“Many rare conditions are life-threatening, and many do not have treatments,” said Sarah Yim, director of the FDA’s Office of Therapeutic Biologics and Biosimilars. “The FDA is committed to facilitating the development of safe and effective interchangeable biosimilar treatments that can expand access for individuals with rare diseases whose current treatment options are limited.” Rare diseases affect fewer than 200,000 people in the U.S. PNH and aHUS are rare diseases characterized by the breakdown of red blood cells, leading to severe health issues.

Bkemv is a monoclonal antibody that binds to the complement C5 protein, inhibiting activation of the complement system, a part of the body’s immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS. However, Bkemv has a Boxed Warning about the increased risk of serious and life-threatening meningococcal infections. Patients must complete meningococcal vaccination before starting Bkemv or Soliris and be monitored for early signs of infections.

As an interchangeable biosimilar, Bkemv is highly similar to Soliris with no clinically meaningful differences. It has the same safety warnings and is expected to have the same adverse reactions as Soliris. The most common adverse reactions in PNH and aHUS trials include headache, nasopharyngitis, back pain, nausea, diarrhea, hypertension, and upper respiratory infections. Bkemv is available only through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS), ensuring that its benefits outweigh its risks.

Rare Diseases

Boosting Rare Diseases Treatment Options

Bkemv is the 53rd approved biosimilar in the U.S., with 13 of these approved as interchangeable biosimilars. An interchangeable biosimilar meets additional requirements under the law, allowing substitution for the reference product without consulting the prescriber, subject to state pharmacy laws. This practice, known as “pharmacy-level substitution,” is similar to how generic drugs are substituted for brand name drugs. The approval of biosimilar and interchangeable biosimilar products supports the FDA’s commitment to a competitive marketplace, increasing patient access to more affordable treatments.

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The FDA’s approval of Bkemv for Amgen Inc. represents a significant advancement in reducing healthcare expenditures while maintaining high standards of safety and efficacy. The introduction of interchangeable biosimilars like Bkemv can lead to substantial cost savings for the healthcare system and improve access to critical treatments for patients with rare diseases.



Resource: U.S. Food and Drug Administration, May 28, 2024

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