WILMINGTON, Del.--(BUSINESS WIRE)--VOYDEYA™ (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH).1 VOYDEYA is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor.2,3. The approval by the US Food and Drug Administration (FDA) was based on positive results from the pivotal ALPHA Phase III trial. Results from the 12-week primary evaluation period of the trial were published in The Lancet Haematology.2. Bart Scott, MD, Professor, Division of Hematology and Oncology at the University of Washington Medical Center, and Professor, Clinical Research Division at Fred Hutchinson Cancer Center, said: “The approval of VOYDEYA offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with ULTOMIRIS or SOLIRIS. Terminal complement inhibition with ULTOMIRIS can address the life-threatening complications of PNH, building on the efficacy and safety of SOLIRIS established over nearly 20 years.”

WILMINGTON, Del.--(BUSINESS WIRE)--ULTOMIRIS® (ravulizumab-cwvz) has been approved in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).1. The approval by the US Food and Drug Administration (FDA) was based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology.2 In the trial, ULTOMIRIS was compared to an external placebo arm from the pivotal SOLIRIS® PREVENT clinical trial. ULTOMIRIS met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Zero relapses were observed among ULTOMIRIS patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%, hazard ratio (95% CI): 0.014 (0.000, 0.103), p0.0001).2

Sebelipase alfa (Kanuma®▼) recommended by NICE as the first treatment for infants with ultra-rare Wolman Disease.  . Kanuma, an enzyme replacement therapy, is the first medicine to be recommended for this life-threatening condition in infants under two years old