Novartis has announced that the FDA Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of children and young adults with B-cell acute lymphoblastic leukemia (ALL) that has relapsed or resisted treatment (r/r). A Biologics License Application (BLA) for this […]

Alnylam and Sanofi Genzyme have announced new positive results from the ongoing Phase 2 study with fitusiran in patients with hemophilia A and B, with or without inhibitors. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT […]

Novartis has announced topline results from the global Phase 3 CANTOS study investigating the efficacy, safety and tolerability of ACZ885 (canakinumab) in combination with standard of care in people with a prior heart attack and inflammatory atherosclerosis. With more than 10,000 patients enrolled in the study over the last six years, CANTOS is one of […]

The FDA is taking two new, important steps to increase competition in the market for prescription drugs and facilitate entry of lower-cost alternatives. The agency published a list of off-patent, off-exclusivity branded drugs without approved generics, and also implemented, for the first time, a new policy to expedite the review of generic drug applications where […]

Waltham-based Syntimmune, a clinical-stage biotechnology company focused on FcRn biology, has announced a $50 million Series B financing led by Apple Tree Partners, which has committed $48 million to the round, with additional participation from other existing investors. The proceeds will further advance Syntimmune’s clinical development program, which includes two ongoing Phase 1b/2a clinical trials […]

The European Commission (EC) has granted a marketing authorization for Biogen drug Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 5q SMA is the most common form of the disease and represents approximately 95 percent of all SMA cases. Spinraza is the first approved treatment in the European Union (EU) for SMA, […]