Drug approval

MIL62, an injection therapy approved exclusively in China for neuromyelitis optica spectrum disorder (NMOSD), was found to keep most NMOSD patients free of relapses for up to two years, while being generally safe and well tolerated. That’s according to data from the Phase 1b portion of a single-center…

Therapies specifically approved by the U.S. Food and Drug Administration (FDA) for neuromyelitis optica spectrum disorder (NMOSD) are superior to rituximab and other off-label medications at preventing relapses and serious side effects, according to a real-world study in the U.S. The study also showed that among FDA-approved therapies, the…

Ultomiris (ravulizumab), an approved infusion therapy for adults with neuromyelitis optica spectrum disorder (NMOSD), is closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Alexion, AstraZeneca Rare Disease. The company signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA).

Bio-Thera Solutions has closed patient enrollment ahead of schedule for a pivotal Phase 2/3 clinical trial in China of its investigational therapy BAT4406F due to the treatment’s “compelling efficacy” for neuromyelitis optica spectrum disorder (NMOSD). The decision is based on a recommendation from an Independent Data Monitoring Committee…

The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who are positive for antibodies against the aquaporin-4 (AQP4) protein. About 75% of people with NMOSD produce antibodies that bind to AQP4, according to the therapy’s developer, Alexion…

Health Canada has approved Uplizna (inebilizumab-cdon) to treat adults in the country with neuromyelitis optica spectrum disorder (NMOSD) who test positive for disease-driving anti-aquaporin-4 (AQP4) antibodies. The decision adds Canada to the list of nations where the medication is now available. The intravenous or into-the-vein infusion, developed by…

Ultomiris (ravulizumab) is now approved in Canada to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who are positive for antibodies against aquaporin-4 (AQP4). According to a press release from Alexion Canada, which will market Ultomiris in the country, it’s the “first and only” long-acting therapy…

Soliris (eculizumab) has been approved in China to treat adults with neuromyelitis optica spectrum disorder, known as NMO or NMOSD, who test positive for antibodies against the aquaporin-4 (AQP4) protein. The therapy — which works to reduce the risk of relapse and ease inflammation among patients — was approved…

The U.S. Food and Drug Administration (FDA) has asked for modifications to the risk evaluation and mitigation strategy — known as REMS — for Ultomiris (ravulizumab) before approving the therapy for most adults with neuromyelitis optica spectrum disorder (NMOSD). Ultomiris already is available in the U.S. for treating…

AstraZeneca has announced filing a legal challenge to certain aspects of the U.S. Inflation Reduction Act (IRA), arguing that there may be unintended but harmful consequences for people living with rare diseases such as neuromyelitis optica spectrum disorder (NMOSD). It joins a number of other pharmaceutical companies…