The U.S. FDA’s latest draft guidance on gene therapies focuses on nonclinical studies using next-generation sequencing-based methods and bioinformatics to evaluate safety risks associated with off-target editing and loss of genome integrity in human gene-edited products.

Mabwell (Shanghai) Bioscience Co. Ltd. has announced that SST-001 (18F-FD4), an α-synuclein-targeted PET tracer developed by Mabwell’s incubated company Synusight Biotech, has received clinical trial clearance from China’s National Medical Products Administration (NMPA). The planned phase I trial will enroll healthy volunteers, patients with multiple system atrophy (MSA), and patients with Parkinson’s disease.

Glyconex Inc. has received approval from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the initiation of a first-in-human phase I trial of GNX-1021, the company’s lead antibody-drug conjugate (ADC) candidate, in patients with advanced gastrointestinal (GI) cancers.

The U.S. FDA’s latest draft guidance on gene therapies focuses on nonclinical studies using next-generation sequencing-based methods and bioinformatics to evaluate safety risks associated with off-target editing and loss of genome integrity in human gene-edited products.

Everest Medicines Ltd. has agreed to acquire a Singapore-based commercial unit of Hasten Biopharmaceuticals (Asia) Ltd. for $150 million up front, gaining market authorization holder rights to 14 marketed products originally developed by Takeda Pharmaceutical Co. Ltd.

If a recent warning letter is anything to go by, the U.S. FDA could be using the 2013 Drug Supply Chain Security Act as another enforcement tool to shut down unauthorized suppliers by clamping down on the dispensers that purchase their unapproved drug products.

The U.S. FDA sent out a friendly reminder to more than 2,200 sponsors and researchers, associated with more than 3,000 clinical trials, who may be delinquent in disclosing the results of those studies on clinicaltrials.gov.

Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint.

In a move that could give patients quick access to drugs their doctors prescribe, the U.S. CMS is proposing a rule giving insurers no more than 24 hours to respond to urgent prior authorization requests and 72 hours for standard requests. The rule also would require full disclosure of claims denials and appeals outcomes.

The U.S. FDA has accepted for review Telix Pharmaceuticals Ltd’s resubmitted NDA for TLX101-Px (Pixclara, 18F-floretyrosine, 18F-FET), its radiolabeled glioma imaging product for characterizing progressive or recurrent glioma in adult and pediatric patients. The FDA assigned a Sept. 11, 2026, PDUFA date.