预约演示

AstraZeneca's rare disease unit overcomes FDA snub to expand Ultomiris in NMOSD

2024-03-25

临床3期上市批准临床结果并购临床2期

In neuromyelitis optica spectrum disorder, Ultomiris’ current competitors include Roche’s Enspryng and Amgen’s Uplizna. (AstraZeneca)

Neaneuromyelitis optica spectrum disorderor Soliris in neuromyelitis optica spectrRochesorder (NMOSD), AmgenZeneca’s follAstraZenecaug Ultomiris has entered the rare neurological disease, as well.

The FDA has cleared UltomirFDAfor patients wSolirisi-AQneuromyelitis optica spectrum disorder (NMOSD)maAstraZenecautes about 75% of allUltomirisses.neurological disease

CompFDAd with SoliriUltomirisris reduces the dosing frequeantibody-positive NMOSDions to once every eight weeks during the maintenance pNMOSD AZ has been hoping that Ultomiris’ long-acting nature could help it reach more patients with various autoimmune diseases.

The NMOSD nod SoliristeUltomirispected. The FDA first rejected Ultomiris in the indication in September, requesting AZ to modify the design of a mandated safety monitUltomirisgram known as a Risk Evaluation and Mitigation Strategy.autoimmune diseases

In NMOSD, Ultomiris’ current competitors incFDAe Roche’s EnspryUltomirise-monthly injection that can be self-administered; and Amgen’s Uplizna, which is an infusion that can be given every six months.

NMOSD is aUltomirisonic autoimmune disorder of thRochetraEnspryngs system affecting about 200,000 people worldwide. It’s more cAmgen inUpliznabeginning in their 30s and 40s.

NMOSDiris provedchronic autoimmune disorder of the central nervous systemIn the study, no patients experienced an adjudicated relapse by the time the last enrolled participant completed their 50-week visit, or after a median treatment duration of 73 weeks. Compared to external control, Ultomiris led to a 98.6% reduction in the risk of relapse.

Ultomirisison, 89% of anti-AQP4 antibody-positive patients who took Amgen’s Uplizna in the N-MOmentum trial remained relapse-free during a six-month period. For Roche’s Enspryng, the relapse-free rate at 96 weeks was 76.5% in the SAKuraStar monotherapy study and 91.1% in the SAkuraSky sUltomiriswhich patients also received baseline immunosuppressant therapy.

With Ultomiris, AZ now offers a treatment option that has the potentAmgeno eUpliznae relapses with a dosing schedule every eight weeks, Marc Dunoyer, CEO of AZ’sRocheionEnspryngsease unit, said in a statement Monday.

FirstUltomiris by the FDA in late 2018, Ultomiris is also allowed in multiple other autoimmune diseases, including generalized myasthenia gravis (gMG) thanks to a 2022 approval. Roche last week just reported a positive phase 3 trial for Enspryng in gMG but said the size of clinical benefit didn’t meet the company’s expectations.

With just the NMOSD laFDA, Enspryng brouUltomiris 256 Swiss francs ($285 million) inautoimmune diseases Uplizna, a generalized myasthenia gravisas part of its $27.8 billion acquiRochen of Horizon Therapeutics, generated $65 million in saEnspryng year after an FDA NMOSD nod in mid-2020. A phase 3 readout for Uplizna in myasthenia gravis is expected in the second half of this year.

Thanks to its multiple indiEnspryng UltomiriRocheled in nearly $3 billion in sales last year, up 51% yeaUpliznayear, although someAmgenhe growth came at the cost of Soliris.Horizon Therapeutics Uplizna myasthenia gravis

AZ is not done expanding Ultomiris. Ultomiris 3 trials are testing the long-acting C5 inhibitor in hematopoietic stem cell transplant-associated thrombotic microangiSolirisand cardiac surgery-associated acute kidney injury. AZ believes both diseases hold blockbuster opportunities.

Besides those studies, miUltomirisials are underway in lupus nephritis and immunoglC5 inhibitor C5ulin A nephropathy. The British pharma suffered a setback rethrombotic microangiopathye 2 trial for Ultomiris in dermaacute kidney injuryisappointing efficacy.

更多内容，请访问原始网站

文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。

机构