(Sharecast News) – Pharma giant AstraZeneca said it is “working closely” with US regulators to clarify some details of its Ultomiris drug as it hopes to bring the treatment to market as quickly as possible for people suffering from the rare disease, neuromyelitis optica spectrum disorder (NMOSD).
Ultomiris is already approved for the treatment of certain adults with NMOSD in the EU, Japan and other countries, but is only available in the US for a select few – “adults with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) Ab+, and certain adults and children with paroxysmal nocturnal haemoglobinuria (PNH) or atypical haemolytic uraemic syndrome (aHUS),” the company explained.
AstraZeneca had submitted a biologics license application to the US Food and Drug Administration (FDA) to approve Ultomiris in the treatment of adults with NMOSD who are anti-aquaporin-4 (AQP4) antibody positive (Ab+).
In a statement on Wednesday, the company said: “The FDA requested modifications to enhance the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment.”
AstraZeneca said it “remains committed to bringing Ultomiris to people living with NMOSD in the US as quickly as possible”, and will work with the FDA regarding the next steps for the REMS modifications.
Positively, the FDA did not ask for additional analysis of trial data or raise any concerns about the drug’s efficacy or safety.
AstraZeneca shares were down 1% at 10,630p by 0812 BST.