Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Atyr, Cidara, GSK, Kite, Kymera, Mindset.
Japan’s Pharmaceutical and Medical Devices Agency has cleared Healios K.K. and Sumitomo Pharma Co. Ltd. to begin a phase I/II study of HLCR-011, which is composed of retinal pigment epithelial (RPE) cells derived from allogeneic induced pluripotent stem cells (iPS) in patients with RPE tear.
Intercept Pharmaceuticals Inc.’s second attempt to score an expanded U.S. FDA approval of its farnesoid X receptor agonist, obeticholic acid, in patients with non-alcoholic steatohepatitis (NASH) went the way of the first, with the agency issuing another complete response letter (CRL), prompting the company to drop all NASH-related investment and cut a third of its workforce.
Sarepta Therapeutics Inc. is set to introduce the first gene transfer therapy for ambulatory patients diagnosed with Duchenne muscular dystrophy (DMD), after the U.S. FDA granted accelerated approval to SRP-9001 (delandistrogene moxeparvovec). Branded Elevidys, the therapy marks Sarepta’s fourth approved treatment for DMD and the first to offer patients a one-time treatment option.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Acelink, Biocardia, Bluebird, Calliditas, Cormedix, Diamedica, Jadeite, Teikoku, Xbrane.
The floodgates have opened for challenges to the new U.S. drug price negotiation process laid out in the Inflation Reduction Act (IRA) that was narrowly passed last year.
Shenzhen Salubris Pharmaceuticals Co. Ltd. received marketing approval from China’s NMPA for enarodustat as a treatment for anemic patients with chronic kidney disease (CKD) that are not on dialysis.
The World Health Organization (WHO) recently gave an emergency use listing (EUL) for SK Bioscience Ltd.’s COVID-19 vaccine called Skycovione, a self-assembled nanoparticle vaccine that targets the SARS-CoV-2 spike protein.
In its newly issued complete response letter (CRL) to Aldeyra Therapeutics Inc., the U.S. FDA said there’s just not enough evidence of efficacy right now to approve ADX-2191, an injectable vitreous-compatible formulation of methotrexate to treat primary vitreoretinal lymphoma (PVRL).
Following a three-month delay, the U.S. FDA approved under priority review a subcutaneous formulation of Argenx SE’s efgartigimod, offering patients a much more convenient way to treat generalized myasthenia gravis (gMG).
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