AstraZeneca PLC (LSE:AZN) said the US Food and Drug Administration (FDA) has responded to a request to expand the use of the drug Ultomiris by asking for a risk evaluation and mitigation strategy.
In doing so, the regulator wants to ensure the benefits of the treatment outweigh the potential downsides. Its request was contained in what's called a complete response letter to data supporting the expanded use of the drug.
Specifically, AZ and its rare diseases business Alexion want to treat a specific group of patients with neuromyelitis optica spectrum disorder (NMOSD), a rare neurological condition.
This group of patients tests positive for a specific antibody called anti-aquaporin-4 (AQP4).
In its reply, the FDA did not ask for more data from the drug's clinical trial or express concerns about its effectiveness or safety.
The FDA's letter is designed to ensure that before patients receive Ultomiris, their vaccination status against a specific infection (meningococcal) is confirmed or they are given preventive antibiotics.
AZ in a stock exchange statement said Alexion is collaborating with the FDA to make these updates. They aim to make Ultomiris available to NMOSD patients in the US soon.
It's worth noting that Ultomiris is already approved for NMOSD treatment in the European Union, Japan and other countries. In the US, the FDA has approved it for other conditions, including generalized myasthenia gravis and certain blood disorders.