Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy.
Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).
Ireland’s Horizon Therapeutics became the first to win regulatory approval in the United States for the treatment of Thyroid Eye Disease (TED), a progressive autoimmune disorder that can threaten the vision of those afflicted.
January 2020 is not a particularly busy month for Prescription Drug User Fee Act (PDUFA) dates for the U.S. Food and Drug Administration, with only two scheduled for the month.
The U.S. FDA issued a Complete Response Letter (CRL) to GlaxoSmithKline’s ViiV Healthcare subsidiary, rejecting an HIV treatment combining cabotegravir and rilpivirine.
The FDA approved BeiGene Ltd.’s lymphoma treatment Brukinsa, validating the China-based drugmaker’s strategy of largely using data from clinical trials held outside the U.S. to file for approval.
The U.S. Food and Drug Administration approved Vumerity (diroximel fumarate) – a novel oral fumarate with a distinct chemical structure – for the treatment of relapsing forms of multiple sclerosis.
Vertex Pharmaceuticals Inc. priced the company’s FDA-approved Trikafta, a three-drug combination for cystic fibrosis, at $311,503 per year.
The U.S. Food and Drug Administration’s Antimicrobial Drugs Advisory Committee voted to recommend approval of Shionogi & Co. Ltd.’s investigational antibiotic cefiderocol for the treatment of complicated urinary tract infections (cUTI), including pyelonephritis, in patients with limited or no alternative treatment options.
Verrica Pharmaceuticals Inc. submitted a New Drug Application to the U.S. Food and Drug Administration for VP-102 (cantharidin 0.7% topical solution) for the treatment of molluscum contagiosum.