Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.
Following a bioresearch monitoring inspection, the U.S. FDA slapped Julio Flamini, a clinical investigator at the Research Center of Atlanta, with a warning letter citing a “significant” protocol violation in which a 15-year-old girl was given 10 times the maximum daily dose of an unnamed study drug for a week.
Distinguishing between contract law and patent law, the U.S. Court of Appeals for the Third Circuit ruled against a Merck KGaA subsidiary, saying Ares Trading SA is still on the hook for paying royalties to a research partner through 2027 on sales of its cancer drug Bavencio (avelumab), a PD-L1 inhibitor granted accelerated approval in 2017 as a treatment for metastatic Merkel cell carcinoma.
Technological breakthroughs are changing the biopharmaceutical landscape and forcing regulators to think on their feet and facilitate (not impede) innovation, experts said at the Global Bio Conference (GBC) 2024. “Regulatory speed and agility are necessary amid emergencies to cater to unmet medical needs,” Choong May Ling, CEO of Singapore’s Health Sciences Authority, told audience members in Seoul, South Korea.
The U.S. FDA is accused of dragging its feet on making public the devices for which it granted market access under the de novo program, and the agency recently been scrambling to bring these decision summaries to light.
Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.
Telix Pharmaceuticals Ltd. filed an NDA with the U.S. FDA for its radiopharmaceutical glioma imaging product, TLX-101-CDx (Pixclara, 18F-floretyrosine, 18F-FET), for the characterization of progressive or recurrent glioma from treatment-related changes in both adult and pediatric patients.
The European Court of Justice declared that the European Commission was out of bounds in attempting to thwart Illumina Inc.’s proposed reacquisition of Grail Inc., which Illumina said relieves it of a fine of roughly €430 million (US$474.92 million).
The U.K. National Institute for Health and Care Excellence sees some evidence that single-step insertions of scaffolds for knees with damaged cartilage offer significant benefits, calling for the use of registries for tracking of outcomes data.
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