AQP4

Alexion is exploring the potential of Voydeya as a monotherapy. Credit: Sai Thaw Kyar/Shutterstock.com. Alexion, AstraZeneca Rare Disease has received approval from the US Food and Drug Administration (FDA) for Voydeya (danicopan) as an add-on therapy to treat extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH). A first-in-class, oral Factor D inhibitor, Voydeya has been developed for patients who continue to suffer from EVH despite treatment with C5 inhibitors such as Ultomiris (ravulizumab) or Soliris (eculizumab). The FDA’s decision is grounded in the positive outcomes of the ALPHA Phase III trial, which demonstrated that Voydeya met its primary endpoint of haemoglobin change from baseline to week 12, alongside all key secondary endpoints. The drug was generally well-tolerated with no new safety concerns. As part of the ALPHA double-blind, placebo-controlled, multiple-dose trial, patients were enrolled and randomised to receive Voydeya or placebo apart from their ongoing Soliris or Ultomiris therapy over 12 weeks. See Also: US FDA grants fast track status to LISCure’s LB-P8 drug for PSC FDA extends review period for Applied Therapeutics’ govorestat NDA It aimed to assess the superiority of Voydeya as an adjunct to C5 inhibitor therapy. The trial included a prespecified interim analysis and an option for a two-year extension period for those completing the initial 24 weeks. Japan already approved Voydeya and the European Union is poised to follow suit. The drug has also achieved breakthrough therapy designation in the US and PRIority MEdicines (PRIME) status in the EU. Alexion CEO Marc Dunoyer stated: “The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science. “As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.” Alexion is also exploring Voydeya’s potential as a monotherapy for geographic atrophy in a Phase II clinical trial. The FDA recently approved Alexion’s Ultomiris to treat adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.

AstraZeneca’s rare disease arm Alexion scored its fourth approval for Ultomiris in a rare autoimmune disorder after Phase 3 data showed there were zero patients with relapses over 73 weeks. The FDA approved Ultomiris, the first and only long-acting C5 complement inhibitor, for the treatment of adult patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease that affects the central nervous system and can relapse unpredictably. Symptoms can include blindness and neurological problems that can lead to permanent disabilities. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

AstraZeneca’s Ultomiris (ravulizumab-cwvz) gained approval from the FDA for the treatment of adults with AQP4 antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The agency had rejected the long-acting C5 complement inhibitor in this indication last year, calling for modifications to the risk evaluation and mitigation strategy (REMS).Specifically, the FDA requested changes to the REMS to further validate patients' meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment. AstraZeneca said Monday that approval of Ultomiris was based on findings from the Phase III CHAMPION-NMOSD trial. The study achieved its primary endpoint of time to first on-trial relapse, with no relapses observed among Ultomiris patients with a median treatment duration of 73 weeks.Marc Dunoyer, CEO of the company’s Alexion unit, noted that Ultomiris “has the potential to eliminate relapses with a convenient dosing schedule every eight weeks.” The intravenous therapy is currently approved to treat AQP4 Ab+ NMOSD in the EU, Japan and Canada, with regulatory reviews ongoing in additional countries.In addition, Ultomiris is authorised in the US for the treatment of: certain adults with generalised myasthenia gravis; certain adults and children with paroxysmal nocturnal haemoglobinuria; and certain adults and children with atypical haemolytic uraemic syndrome to inhibit complement-mediated thrombotic microangiopathy.