Although PDUFA VI still has two years of life left to it, PDUFA VII is already in the birthing process, with the use of real-world data (RWD), AI, and a coming surge of novel cell and gene therapies looking to be prominent features of the next five-year user fee agreement. Politics likely will play a role as well.
The FDA has lifted all the clinical holds it placed on seladelpar from Cymabay Therapeutics Inc. for INDs in nonalcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC) and primary sclerosing cholangitis, giving the company new hope.
The FDA has approved Xywav (calcium, magnesium, potassium and sodium oxybates; JZP-258), an oxybate product for treating both cataplexy and excessive daytime sleepiness in narcolepsy patients ages 7 and older, from Jazz Pharmaceuticals plc.
Interludes of classical music. Little kids talking in the background. Unmuted mics as panelists multitask. Gurgles of “underwater” sound. Periods of silence as speakers forget to unmute. And then the technical problems – lots of them. Such are the challenges of addressing a virtual FDA advisory committee in the time of COVID-19.
With an Aug. 22 PDUFA date set, Tricida Inc. said it received an FDA notification regarding the NDA for veverimer (TRC-101) for treating metabolic acidosis in chronic kidney disease (CKD) patients, noting that it had identified deficiencies precluding discussion of labeling and postmarketing requirements/commitments.
While members of the FDA’s Cardiovascular and Renal Drugs Advisory Committee all recognized the severity of hepatorenal syndrome type 1 (HRS-1) and the significant unmet need in the space, they were divided on whether the FDA should follow the decades-old lead of regulators in 60 other countries in approving Mallinckrodt plc’s terlipressin as an HRS-1 treatment.
Patients with triple-refractory multiple myeloma who are running out of options may soon have a new lifeline. That’s if the FDA follows the unanimous vote of its Oncologic Drugs Advisory Committee (ODAC) and grants accelerated approval to Glaxosmithkline plc’s (GSK) belantamab mafodotin as a fifth-line therapy for adults with relapsed or refractory multiple myeloma.
Two subcommittees of the U.S. House Appropriations Committee have moved their respective appropriations proposals for the FDA and the NIH, restarting a process that has worked smoothly over the past couple of years. Still, Republicans in both committees objected to the use of emergency funding mechanisms in lieu of more routine appropriations.
A month ahead of its PDUFA date, Ultragenyx Pharmaceutical Inc.’s NDA for triheptanoin (UX-007) won FDA approval. Branded Dojolvi, the highly purified, synthetic, seven-carbon fatty acid triglyceride was cleared for use in treating pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
The push for a vaccine for the COVID-19 pandemic may have no parallel in pharmaceutical history, and FDA commissioner Stephen Hahn vowed in a Senate hearing that the agency will rely on the agency’s traditional standards for scientific evidence in premarket reviews of those vaccines.
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