Pacira BioSciences PCRX announced that the Centers for Medicare and Medicaid Services (CMS) has issued a permanent product-specific J-code (J0666) for its lead pain-management drug, Exparel (bupivacaine...
Zevra Therapeutics, Inc. ZVRA obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC) in September. The drug will be marketed under the brand name Miplyffa.The...
Ultragenyx Pharmaceutical RARE reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study evaluating UX701, an investigational AAV9 gene therapy, to treat Wilson disease. Stage...
Ionis Pharmaceuticals, Inc. IONS announced that the FDA has granted a Fast Track designation to its investigational RNA-targeted therapy zilganersen for treating Alexander disease (AxD), an ultra-rare...
Cassava Sciences SAVA has agreed to pay a $40 million fine to settle charges from the U.S. Securities and Exchange Commission (SEC) over allegedly misleading statements about the results of its 2020 mid-stage...