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The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA) and making a final decision on marketing approval. The typical period for review is 10 months after the drug application has been accepted by the Agency. For drugs that have Priority Review, the review period is reduced to 6 months from the time of application acceptance.
Vonoprazan for Erosive Gastroesophageal Reflux Disease (GERD)
PDUFA date: November 17, 2023
The FDA is reviewing the resubmitted New Drug Application (NDA) for vonoprazan, an oral small molecule potassium-competitive acid blocker, for the treatment of erosive GERD. The application is supported by data from the double-blind phase 3 PHALCON-EE trial (ClinicalTrials.gov Identifier: NCT04124926), which compared the efficacy and safety of vonoprazan to lansoprazole in 1024 patients with erosive GERD. The resubmitted NDA also includes 3 months of stability data for 6 batches of the reformulated vonoprazan tablet to support the commercial shelf life of vonoprazan.
Valneva’s Chikungunya Virus Vaccine Candidate VLA1553
PDUFA date: November 2023
Chikungunya is a mosquito-borne viral disease caused by the chikungunya virus, a Togaviridae virus. VLA1553 is a live-attenuated, single-dose vaccine designed to delete a part of the chikungunya virus genome. The BLA is supported by data from a double-blind, randomized, phase 3 trial (ClinicalTrials.gov Identifier: NCT04546724) that evaluated the immunogenicity and safety of VLA1553 in 4128 healthy adults 18 years of age and older. A regulatory decision was expected in August 2023, however, the FDA extended the target date by 3 months to allow for sufficient time to discuss the details of a confirmatory trial.
Nirogacestat for the Treatment of Desmoid Tumors
PDUFA date: November 27, 2023
Desmoid tumors, also known as aggressive fibromatosis or desmoid-type fibromatosis, are rare, generally nonmalignant, soft tissue tumors that are abnormal growths of connective tissue. Nirogacestat is an oral, selective, small molecule gamma secretase inhibitor that works by blocking proteolytic activation of Notch receptors. The NDA for nirogacestat was accepted for Priority Review based on data from the phase 3 DeFi trial (ClinicalTrials.gov Identifier: NCT03785964). Findings demonstrated that treatment with nirogacestat significantly reduced the risk of disease progression by 71% compared with placebo.
Reproxalap for the Treatment of Dry Eye Disease
PDUFA date: November 23, 2023
Reproxalap is a first-in-class reactive aldehyde species (RASP) modulator that covalently binds to free aldehydes and diminishes excessive RASP levels, which are generally elevated in ocular and systemic inflammatory disease. The NDA is supported by efficacy and safety data from 5 clinical trials, including the phase 3 TRANQUILITY-2 study (ClinicalTrials.gov Identifier: NCT05062330), which included 361 patients with dry eye disease. Findings showed reproxalap was statistically superior to vehicle for the primary endpoint (overall mean change from baseline in Schirmer test, a measure of ocular tear production).
TAK-755 for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura
PDUFA date: November 2023
Congenital thrombotic thrombocytopenic purpura results from a deficiency in ADAMTS13, a von Willebrand factor (VWF) cleaving protease. This leads to an accumulation of ultra-large VWF multimers in the blood and uncontrolled platelet aggregation and adhesion. TAK-755 is a recombinant ADAMTS13 protein designed to replace the missing or deficient ADAMTS13 enzyme. The BLA includes data from a randomized, controlled phase 3 study (ClinicalTrials.gov Identifier: NCT03393975). Findings showed that the incidence of thrombocytopenia events was reduced with TAK-755 compared with standard of care therapy.
