When it won U.S. FDA accelerated approval more than eight years ago, Intercept Pharmaceuticals Inc.’s Ocaliva (obeticholic acid) was viewed as a breakthrough, becoming the first new treatment in 20 years for rare, progressive liver disease primary biliary cholangitis (PBC) and, for several years, the only second-line treatment for PBC patients failing to respond to ursodeoxycholic acid. More recently, however, Ocaliva has faced regulatory and safety stumbles, with Intercept now voluntarily pulling the farnesoid X receptor activator from the U.S. market.
The U.S. FDA issued new guidance for the development of non-opioid analgesics for chronic pain indications, with specific details on trial design, patient populations and meaningful outcomes, including reducing the nation’s reliance on opioids.
“The comment I hear a lot from scientists … is that science has no borders,” Arif Noorani, partner at Sidley Austin LLP, said while addressing the panel audience at Asia Bio 2025 in Singapore. “I agree, but the reality is, we do have a lot of borders.”
It took a memo from the president for the U.S. FDA to begin reining in direct-to-consumer prescription drug advertising with its feel-good messaging and hurried recitation of a few serious adverse events.
Less than two months after receiving priority review status for an NDA, Johnson & Johnson won U.S. FDA approval of Inlexzo, its intravesical gemcitabine-releasing system previously known as TAR-200, to treat adults with Bacillus Calmette-Guérin-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ, with or without papillary tumors.
Several South Korean biotech and biopharmaceutical companies completed IND submissions or won nods to start clinical trials in either the U.S. or South Korea, including SK Bioscience Co. Ltd., Genosco Inc., Pimedbio Inc., Sillajen Inc. and Ami Pharm Co. Ltd.
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s rare disease treatment, sodium dichloroacetate (SL-1009), for pyruvate dehydrogenase complex deficiency. The inhibitor of pyruvate dehydrogenase kinases is the only drug in development for treating the rare genetic disorder, according to Cortellis. There are no FDA-approved treatments for the disease.
Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced the granting of orphan drug designation by the FDA to BA-101 for the treatment of glioblastoma (GBM). The company is advancing development of BA-101 toward first-in-human studies.
Sometimes little things can create big problems, which seems to be the case with the recall of the power unit used with the Abbott Heartmate 3 left ventricular assist device (LVAD). According to the FDA recall announcement, the power cords used with the power supply might not stay fixed in place, which would leave the power supply with 15 minutes of backup battery runtime before depletion.
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