Voydeya (danicopan), from Alexion, Astrazeneca Rare Disease, racked up its second global approval as the U.S. FDA greenlit it as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH), a crowded market with several already approved treatments and more in development.
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
A prespecified interim analysis revealed that Astrazeneca plc’s oral factor D inhibitor danicopan met the primary endpoint of a phase III trial as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who were also taking a C5 inhibitor, but who still experienced extravascular hemolysis.
Dianthus Therapeutics Inc. has, appropriately, flowered in springtime. The Waltham, Mass.-based company emerged from stealth with $100 million in series A funding and lofty ambitions to rewrite the rules of targeting the complement system with a pipeline of antibodies that bring new levels of selectivity to an area of innate immunity that has proved difficult to target.
As it settles a patent dispute with Japan’s Chugai Pharmaceutical Co. for $775 million, the U.K.’s Astrazeneca plc is looking to bring intellectual property in-house by snapping up a new generation of talented scientists.
As it settles a patent dispute with Japan’s Chugai Pharmaceutical Co. for $775 million, the U.K.’s Astrazeneca plc is looking to bring intellectual property in-house by snapping up a new generation of talented scientists.
A month after Astrazeneca plc’s $39 billion takeover of Alexion Pharmaceuticals Inc. was finalized, the merged company has hit its first speed bump after the new unit discontinued a phase III rare disease trial. Alexion, of Boston, said it axed the 382-patient CHAMPION-ALS trial of its long-acting C5 complement inhibitor Ultomiris (ravulizumab) in amyotrophic lateral sclerosis (ALS).
Citing a lack of efficacy from adding its complement inhibitor Ultomiris (ravulizumab) to best supportive care for patients hospitalized with severe COVID-19, Alexion Pharmaceuticals Inc. paused further enrollment in a global phase III study of the drug. The move, recommended by the trial's independent data monitoring committee, suggested Ultomiris may soon join the list of other therapeutics once tested against COVID-19 but now no longer.
Alexion Pharmaceuticals Inc., of Boston, will diversify its portfolio beyond its top-selling Soliris (eculizumab) and C5 inhibitor Ultomiris (ravulizumab) by acquiring Portola Pharmaceuticals Inc. for $1.4 billion. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Alexion’s CEO, Ludwig Hantson, told investors May 5 that the acquisition immediately diversifies the company’s commercial-stage portfolio by adding Portola’s Andexxa (coagulation factor Xa [recombinant], inactivated-zhzo) to Alexion’s existing palette of hematology and neurology therapies. Andexxa is the only FDA-approved factor Xa inhibitor reversal agent in severe and uncontrolled bleeding.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”