Shares in Ardelyx Inc. have crashed after its tenapanor, under FDA review for managing phosphate levels in chronic kidney disease (CKD), ran into trouble with the regulator. The company said that the FDA has identified “deficiencies” in the data package, which prevent discussions about labeling and postmarketing commitments.
Despite an FDA advisory committee’s narrow ruling that Provention Bio Inc.’s teplizumab has benefits that outweigh the risks in treating diabetes, the agency sent the company a complete response letter (CRL) regarding the diabetes therapy’s priority BLA. The CRL came the evening of July 2, Provention said, the PDUFA date that had been set months before.
PERTH, Australia – Cyclopharm Ltd. said it received a complete response letter (CRL) from the FDA for its Technegas NDA for pulmonary embolisms. The CRL relates to better defining and validating production and delivery of the Technegas particle and other aspects of crucible manufacturing and dosimetry that need to be addressed before the NDA can be approved.
A not-unexpected complete response letter (CRL) for Nexobrid, being developed by Mediwound Ltd., delayed the therapy’s approval and knocked the company’s share values backward. Nexobrid, proteolytic enzymes enriched in bromelain for treating for eschar removal (debridement) in adults with deep partial-thickness and/or full-thickness thermal burns, had a June 29 PDUFA date.
Shares in Orphazyme A/S cratered after the FDA rejected its arimoclomol for Niemann-Pick disease type C, a rare and potentially fatal inherited condition where fat builds in tissues and organs.
Alkermes plc worked its way through a complete response letter (CRL) issued in late 2020 as the FDA has approved Lybalvi (olanzapine and samidorphan) for treating adults with schizophrenia and bipolar I disorder. The once-daily, oral antipsychotic treatment, the company’s first, is for maintenance monotherapy or acute treatment of manic or mixed episodes and as a monotherapy or adjunct to lithium or valproate. The therapy had a June 1 PDUFA date.
Shares of Protalix Biotherapeutics Inc. (NYSE:PLX) fell 33% to $3.93 on April 28 after the company reported receiving an FDA complete response letter (CRL) in answer to its BLA for pegunigalsidase alfa, a galactosidase enzyme replacement therapy it has long advanced for the potential treatment of Fabry disease.
Acadia Pharmaceuticals Inc. President Serge Stankovic said the company was “kept completely in [the] dark” by the FDA regarding specifics about what the agency found wrong a month ago with the sNDA for Nuplazid (pimavanserin) – until, that is, the agency’s complete response letter (CRL) landed on the firm’s desk.
In a move that caught analysts and investors by surprise, the FDA rejected Athenex Inc.’s NDA for Oraxol, its oral version of paclitaxel, for use in metastatic breast cancer, citing safety risks and questioning the reliability of the clinical data supporting the application.
Osmotica Pharmaceuticals plc’s amendment earlier this month to the NDA for arbaclofen in spasticity resulting from multiple sclerosis wasn’t enough to quell the concerns of the FDA, which smacked the firm with its second complete response letter (CRL).
