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.
Roche Holding AG’s investigational monoclonal antibody, crovalimab, which inhibits part of the innate immune system in patients with the rare blood condition paroxysmal nocturnal haemoglobinuria, met its co-primary efficacy endpoints in a phase III trial that will form the basis of its approval submissions worldwide.
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.
Canada’s Federal Court of Appeal took the Patented Medicine Prices Review Board to task for ordering Alexion Pharmaceuticals Inc. to “forfeit excess revenues” generated by Soliris (eculizumab) between 2009 and 2017.
The FDA’s go-ahead for Roche Holding AG’s Enspryng (satralizumab-mwge) in anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder had watchers of the space weighing its market odds against two therapies approved earlier: Soliris (eculizumab) from Boston-based Alexion Pharmaceuticals Inc., and the more recently cleared Uplizna (inebilizumab-cdon) from Viela Bio Inc., of Gaithersburg, Md.
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.”
Approval of Alexion Pharmaceutical Inc.'s Soliris (eculizumab) injection to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive makes it the first and only FDA-approved treatment. It's also one of the most expensive treatments in the world, making it vulnerable to off-label use and the eventual creep of biosimilars into the market.