The U.S. FDA reported a pilot program for validation of lab-developed tests (LDTs) used as companion diagnostics, a move that seems an implicit recognition that test kits as CDx products are not the darlings of test developers. The program arrives as the agency is considering rulemaking for regulation of LDTs, however, a combination of developments that promises to roil the already strained relationship between the FDA and clinical labs.
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.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Eyenuk, Haemonetics, VVT Medical.
The question of when U.S. federal attorneys can dismiss a whistleblower suit filed under the False Claims Act (FCA) has roiled the courts for several years, but the Supreme Court has laid many of those questions to rest in an 8-1 ruling which said that the government can dismiss a whistleblower FCA case only after federal attorneys have intervened.
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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