FDA Approves Amgen's Soliris Biosimilar Bkemv for Rare Blood Diseases

The FDA announced its approval of Amgen's Bkemv (eculizumab-aeeb) on Tuesday, marking it as the first interchangeable biosimilar to AstraZeneca’s Soliris (eculizumab). This approval pertains to the treatment of two rare blood disorders: paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS). Bkemv, a monoclonal antibody targeting C5, functions by inhibiting the complement system to prevent intravascular haemolysis, effectively managing PNH and aHUS. However, Bkemv's approval does not cover two other conditions treated by Soliris: generalized myasthenia gravis and neuromyelitis optica spectrum disorder.

In Europe, Amgen's biosimilar received approval for PNH last year and is marketed under the name Bekemv. The FDA emphasized Bkemv's high similarity to Soliris, citing "no clinically meaningful differences" between the two, as indicated by the Phase III DAHLIA study results from 2022. Additionally, the FDA confirmed Bkemv's compliance with other legal criteria, allowing it to be considered interchangeable with Soliris at the pharmacy level.

Sarah Yim, director of the FDA’s office of therapeutic biologics and biosimilars, highlighted the significance of such approvals. She noted that interchangeable biosimilars like Bkemv could broaden access to treatment for individuals with rare diseases who currently have limited options. Similar to Soliris, Bkemv will include a boxed warning about the increased risk of serious and life-threatening meningococcal infections caused by Neisseria meningitidis.

Soliris, which became part of AstraZeneca's portfolio following its $39-billion acquisition of Alexion Pharmaceuticals in 2021, is on track to lose its US exclusivity in 2025 due to a patent settlement between Alexion and Amgen. Soliris generated nearly $3.15 billion in global sales last year, underscoring its financial significance to AstraZeneca.

In summary, the FDA's approval of Bkemv as an interchangeable biosimilar to Soliris represents a notable advancement in the treatment of rare blood disorders, potentially enhancing patient access to necessary therapies.