Refusal to File letter Archives

FDA Action Alert: Jazz, ANI and Ultragenyx

Approvals, Cataplexy, Excessive Daytime Sleepiness, Fatty Acids, FDA, FDA/Regulatory, Gels, Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD), Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD), Multiple Sclerosis, Narcolepsy, New Drug Applications, PDUFA, Refusal to File letter, Rheumatoid Arthritis, Supplemental New Drug Application (sNDA), Systemic Lupus Erythematosus (SLE), Ulcerative Colitis

U.S. Food and Drug Administration PDUFA dates on the calendar for July include an NDA review for Jazz Pharmaceuticals’ experimental medicine for cataplexy.

FDA Declines Review of Bristol Myers Squibb and bluebird’s Multiple Myeloma Therapy

B-cell maturation antigen (BCMA), Biologics License Application (BLA), Business, CAR-T Therapy, Collaborations, FDA, FDA/Regulatory, Immunotherapies, Investors, Refusal to File letter, Relapse/Refractory Multiple Myeloma, Shareholders, Shares

Shares of bluebird bio were down in trading after the U.S. Food and Drug Administration declined to review a Biologics License Application for the experimental CAR-T treatment idecabtagene vicleucel for multiple myeloma co-developed with Bristol Myers Squibb.

Zogenix’s seizure drug filing fails to pass U.S. FDA scrutiny

Dravet syndrome, Epilepsy, FDA, FDA/Regulatory, Investors, Refusal to File letter, Seizures, Shares

The FDA refused to fully review the marketing application for Zogenix’s treatment for seizures associated with Dravet syndrome.