AstraZeneca's (AZN) Ultomiris Gets FDA Nod for New Indication
AstraZeneca AZN announced that the FDA has approved its drug Ultomiris for a new rare neurological disease.
The approval for long-acting C5 complement inhibitor Ultomiris is for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). The approval makes Ultomiris the first and the only long-acting C5 complement inhibitor approved for AQP4 Ab+ NMOSD.
Patients with NMOSD experience unpredictable relapses, which can involve the onset of new neurological symptoms or the exacerbation of existing ones. These relapses tend to be severe and recurrent, often leading to permanent disability in affected individuals.
The approval of Ultomiris for AQP4 Ab+. NMOSD was based on data from the CHAMPION-NMOSD phase III study, which demonstrated that Ultomiris led to unprecedented relapse risk reduction in NMOSD patients. Zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks.
In September last year, the FDA issued a complete response letter (CRL) to its supplemental biologics license application (sBLA) seeking approval for Ultomiris for the treatment of adult patients with AQP4 Ab+. NMOSD. However, the FDA did not request any additional analyses of data from the CHAMPION-NMOSD phase III study. It also did not raise any concerns about the efficacy and safety data from the study. The CRL rather requested modifications in the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) safety program.
In the past year, the stock has declined 4.2% against the industry’s 26.9% rise.
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Ultomiris is already approved for the AQP4 Ab+ NMOSD indication in the EU and in Japan.
In the United States, Ultomiris is presently approved for treating three indications — generalized Myasthenia Gravis (gMG), paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Ultomiris was added to AstraZeneca’s portfolio with the July 2021 acquisition of Alexion. The drug recorded sales of $2.97 billion in 2023, up 52% at constant exchange rates. Ultomiris sales are benefiting from label expansion for the gMG indication, expansion into new markets and continued conversion from Alexion’s older drug, Soliris.
Zacks Rank & Stocks to Consider
Currently, AstraZeneca has a Zacks Rank #3 (Hold).
AstraZeneca PLC Price and Consensus
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