The U.S. FDA cleared Paragonix Technologies Inc.’s Kidneyvault portable renal perfusion system, which combines digital monitoring capabilities and hypothermic perfusion technology to preserve donor kidneys during transportation.
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase).
Ideaya Biosciences Inc. has announced FDA clearance of an IND application for IDE-275 (GSK-959), a small-molecule inhibitor of Werner helicase (WRN) discovered by Ideaya in collaboration with GSK plc.
With the U.S. FDA’s approval of Orlynvah (sulopenem etzadroxil and probenecid) for uncomplicated urinary tract infections, Iterum Therapeutics plc can move on from regulatory delays and prepare to launch only the second FDA-approved treatment for the indication in the past 20 years.
Medtronic plc added a second U.S. FDA pulsed field ablation (PFA) device approval to its scorecard with the agency’s greenlight for its Affera mapping and ablation system with the Sphere-9 catheter. Affera brings the first radiofrequency/PFA device to the cardiac ablation market and ups the ante in the PFA competition.
The Medical Information Working Group again took up a question regarding U.S. FDA-regulated speech, citing the agency’s new misinformation draft guidance as an example of a piecemeal approach to regulated speech.
The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.
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