FDA Blocks Approval of AstraZeneca's Ultomiris for Neuromyelitis Optica Spectrum Disorder
Key Insights
The FDA issued a complete response letter for AstraZeneca's Ultomiris (ravulizumab) for neuromyelitis optica spectrum disorder (search) (NMOSD (search)), requesting modifications to the risk evaluation and mitigation strategy (REMS).
The FDA is seeking enhancements to the REMS to ensure patients' meningococcal vaccination status or prophylactic antibiotic administration before Ultomiris treatment.
Ultomiris is already approved for NMOSD (search) in the EU, Japan, and other countries, and AstraZeneca is collaborating with the FDA to address the REMS requirements for US approval.
AstraZeneca's efforts to expand the label for Ultomiris (ravulizumab) have hit a roadblock as the FDA declined to approve its use for neuromyelitis optica spectrum disorder (search) (NMOSD (search)). This decision comes despite Ultomiris already being approved for this indication in the EU, Japan, and other countries.
The FDA's complete response letter (CRL) did not request additional clinical data but instead focused on the risk evaluation and mitigation strategy (REMS) for the drug. The regulator is seeking modifications to the REMS to ensure that patients' meningococcal vaccination status or prophylactic administration of antibiotics are checked before starting Ultomiris treatment. This request stems from data suggesting that meningococcal vaccines may provide incomplete protection against invasive meningococcal disease in patients receiving Soliris and Ultomiris. Current recommendations advise clinicians to consider antimicrobial prophylaxis for these patients to mitigate the risk of meningococcal disease.
AstraZeneca is working closely with the FDA to address the REMS requirements and remains committed to bringing Ultomiris to people living with NMOSD (search) in the US as quickly as possible. Ultomiris is currently FDA-approved for other indications previously held by Soliris, including paroxysmal nocturnal haemoglobinuria (search) (PNH (search)), haemolytic uraemic syndrome (search) (HUS (search)), and generalised myasthenia gravis (search) (gMG (search)).
Ultomiris is a long-acting complement C5 inhibitor designed as a successor to Soliris, offering a more convenient dosing schedule of infusions every eight weeks compared to Soliris's bi-weekly infusions. The transition to Ultomiris is crucial for AstraZeneca, as Soliris is set to lose patent protection and face biosimilar competition in the US in 2025. Several companies, including Amgen, are developing biosimilars for Soliris.
In the NMOSD (search) treatment landscape, Soliris faces competition from Horizon Therapeutics' Uplizna (inebilizumab) and Roche's Enspryng (satralizumab).