UK pharmaceutical company AstraZeneca (AZ; NASDAQ: AZN) has received a rejection from the US Food and Drug Administration (FDA) for an indication extension of its complement inhibitor Ultomiris (ravulizumab) to treat neuromyelitis optica spectrum disorder (NMOSD) in AQP4 antibody positive (Ab+) adults. The FDA has requested modifications to the drug’s safety program, specifically to validate patients’ history of meningococcal vaccination and antibiotics use, before considering the indication extension.
Ultomiris’ Current Approvals and Competitive Landscape
Ultomiris is already approved in the US for the treatment of generalized myasthenia gravis (gMG), atypical haemolytic uraemic syndrome (aHUS), and paroxysmal nocturnal haemoglobinuria (PNH). However, it faces competition in the PNH indication from Roche’s (SWX: RO) investigational drug crovalimab, which is currently under review, according to Fineline Info & Tech data.
Global Market Presence and NMOSD Indications
AstraZeneca’s Ultomiris is present in markets including Brazil, Chile, Ecuador, the European Union (EU), Japan, and Peru, with NMOSD indications approved in Brazil and endorsed in Europe. The rejection in the US for the NMOSD indication does not impact these existing approvals and endorsements, but it does limit the potential expansion of Ultomiris’ use in the US market.-Fineline Info & Tech
