AstraZeneca PLC (LON: AZN) announced that the US Food and Drug Administration (FDA) has requested a risk assessment and mitigation strategy in response to a proposal to broaden the application of the drug Ultomiris.
The regulatory body’s move aims to ascertain that the advantages of the medication surpass its potential risks. The FDA articulated its request through a complete response letter regarding the data supporting the drug’s extended use.
AstraZeneca, along with its subsidiary Alexion specializing in rare diseases, is seeking approval to administer Ultomiris to a particular segment of patients with neuromyelitis optica spectrum disorder (NMOSD), an uncommon neurological ailment. These patients exhibit positive results for a distinct antibody known as anti-aquaporin-4 (AQP4).
In its response, the FDA neither demanded additional clinical trial data nor voiced apprehensions about the drug’s efficacy or safety. Instead, the FDA’s correspondence underscores the importance of verifying patients’ vaccination status against a specific infection (meningococcal) before administering Ultomiris or ensuring they receive preventive antibiotics.
AstraZeneca disclosed in a market update that Alexion is working alongside the FDA on these modifications, aiming to make Ultomiris accessible to NMOSD patients in the US in the near future.
It’s important to highlight that Ultomiris has already received approval for NMOSD treatment in regions like the European Union, and Japan, among others. In the US, while it’s sanctioned for different ailments, such as generalized myasthenia gravis and specific blood-related conditions, its approval for NMOSD is still pending.
