Astellas Pharma’s blood cancer drug Xospata continues to show impressive results in the acute myeloid leukemia setting.
A European Medicines Agency panel recommended a conditional marketing approval for a gene therapy from Bluebird Bio Inc. as a genetic blood disorder treatment, setting the stage for the U.S. biotech to win the company’s first regulatory nod.
Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration granted Breakthrough Therapy designation for Tibsovo (ivosidenib) in combination with azacitidine for the treatment of newly diagnosed acute myeloid leukemia (AML) with an IDH1 mutation in adult patients who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
Denmark-based Novo Nordisk snagged approval from the U.S. FDA for a new treatment for hemophilia A, though Esperoct will not be available in the United States until 2020.
Sanofi reportedly ended 38 R&D programs and is prioritizing oncology, immunology, rare disease and rare blood disorders.
The U.S. FDA approved Cablivi in combination with plasma exchange and immunosuppression for treating acquired thrombotic thrombocytopenic purpura (aTTP) in adults.
Switzerland’s Roche will donate the company’s new hemophilia A drug Hemlibra to a World Hemophilia Federation program, joining rivals who also back the effort to help patients in developing countries who face treatment hurdles.
Novartis’ crizanlizumab receives FDA Breakthrough Therapy designation for prevention of vaso-occlusive crises in sickle cell disease
Novartis announced that the U.S. Food and Drug Administration granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises in patients of all genotypes with sickle cell disease.