The FDA has approved Ionis Pharmaceuticals’ ZANVASTRO for pediatric and adult Alexander disease after a 54-patient study showed significant stabilization of gait at 61 weeks.
Heartvue.ai has received FDA 510(k) clearance for Heartvue.Proton, machine-learning software that automates key cardiac MRI measurements and includes an authorized Predetermined Change Control Plan.
Nyxoah’s ACCCESS pivotal study produced a 77.2% AHI responder rate and 87.7% oxygen-desaturation responder rate with no device-related serious adverse events through 12 months.
Scholar Rock has started dosing in the 60-patient Phase 2 FORGE trial of apitegromab in facioscapulohumeral muscular dystrophy after receiving FDA Fast Track and Orphan Drug designations.
The FDA has accepted United Therapeutics’ supplemental Tyvaso application for idiopathic pulmonary fibrosis after combined TETON data showed a 111.8 mL advantage in forced vital capacity over placebo at 52 weeks.
uniQure has submitted AMT-130 for accelerated U.S. approval after three-year Phase 1/2 data showed a 75% slowing of Huntington’s disease progression against a matched natural-history control.
CMS has approved a New Technology Add-on Payment for Bayesian Health’s FDA-cleared continuous sepsis flagging software, creating an additional Medicare reimbursement pathway for eligible inpatient use beginning October 1.
Hamamatsu has announced U.S. FDA 510(k) clearances for the NanoZoomer S20MD and S540MD whole-slide imaging systems, extending its regulated digital pathology portfolio from compact benchtop use to high-throughput laboratories.
Norroy Bioscience has started pivotal Phase 3 development of 177Lu-NYM032 in PSMA-positive metastatic castration-resistant prostate cancer, with a registered target enrollment of about 600 patients and overall survival as the primary objective.
The ML4 Foundation says the FDA has allowed the first U.S. human trial of an AAV9 gene therapy for mucolipidosis type IV to proceed, opening clinical testing for an ultra-rare neurodegenerative disease with no approved disease-modifying therapy.