The U.S. FDA has approved Duvyzat (givinostat), from Italfarmaco SpA, for treating Duchenne muscular dystrophy (DMD). It is the first oral, nonsteroidal drug for treating all of DMD’s genetic variants. The oral treatment is approved for those ages 6 and older.
A metabolite that suppresses appetite and food intake after exercise could be the reason for the weight loss observed in patients treated with metformin to control blood glucose. A study conducted by a group of scientists at Stanford University showed how this antidiabetic drug induced the biosynthesis of N-lactoyl-phenylalanine (Lac-Phe), which has an effect reducing the body mass index.
Kevin Dills, who the U.S. SEC said secretly controlled Oncology Pharma Inc., consented to a final civil judgment in federal district court related to a fraudulent stock-selling scheme.
Triarm Therapeutics Ltd. is on a mission to democratize CAR T therapies to make them more accessible and affordable, Triarm CEO Jay Zhang told BioWorld. “Nearly half of the patients eligible for CAR T therapies still cannot get treated, and the main reason is the expense, and the second is they cannot afford to wait.
The latest filing from Orlando-based Noble International Inc.—an Aptar Pharma company—hints at the development of a new category of medical devices. Best known for its medical device training solutions and patient onboarding strategies, Noble filed for protection of a compression sleeve for monitoring and treating rheumatic disorders.
The U.S. FDA’s pending final rule for regulation of lab-developed tests has proven unusually controversial even for the FDA, but Rep. Anna Eshoo (D-Calif.) stated in a March 21 hearing that congressional inaction has left the agency in an uncomfortable spot.
An advocate general (AG) for the E.U. Court of Justice did not mince words in calling out the European Commission for its “very significant extension of the scope of the Merger Regulation and of the commission’s jurisdiction” in reviewing Illumina Inc.’s $7.1 billion acquisition of Grail LLC.
As part of its required series of guidances on using real-world evidence, the U.S. FDA released a draft guidance in response to sponsors’ growing interest in the potential use of observational studies to contribute to a demonstration of the effectiveness or safety of a drug or biologic.
Although there’s bipartisan interest in the U.S. Congress to hold pharmacy benefit managers (PBMs) accountable for their contribution to the costliest drug prices in the world, the Biden administration ignored PBMs when it again focused on drug companies as the bad guys of pricing in its proposed 2025 budget.
Two days after the U.S. FDA announced approval of gene therapy Lenmeldy (atidarsagene autotemcel), making it the first treatment option for rare disease metachromatic leukodystrophy, Orchard Therapeutics and parent firm Kyowa Kirin Co. Ltd. disclosed the wholesale acquisition price of $4.25 million for the one-time treatment, which edges out hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to become the world’s most expensive drug.
RSS
