Clinical Trial News

FDA rejected Regeneron's applications for accelerated approval due to the absence of a confirmatory study, signaling potential use of new 2022 authority, possibly facing criticism for delaying serious disease treatments.

Milestone Pharmaceuticals resubmits NDA for etripamil, a calcium channel blocker nasal spray for treating PSVT, addressing FDA concerns. If approved, etripamil could offer a self-administered treatment option for PSVT and AFib-RVR, potentially impacting millions of patients in the U.S.

Aldeyra Therapeutics plans to resubmit a New Drug Application for reproxalap, a RASP modulator for dry eye disease, with a dry eye chamber trial in H1 2024, aiming for NDA resubmission in H2 2024, contingent on trial success.

Enfortumab vedotin plus pembrolizumab significantly improves survival in untreated advanced urothelial cancer, offering a potential first-line alternative to chemotherapy. China's NMPA accepted the sBLA, marking a step towards a new treatment paradigm.

Applied Therapeutics announces FDA extension of review period for govorestat NDA by three months, new PDUFA target action date set for November 28, 2024, for treatment of Classic Galactosemia. Govorestat, an investigational ARI, demonstrated rapid and sustained reduction in galactitol with favorable safety profile in Phase 3 studies. If approved, it would be the first medication for Galactosemia.

Sequana Medical NV, a pioneer in treating fluid overload in liver disease, heart failure, and cancer, has announced its financial results for the year ended 31 December 2023, along with a business update and outlook for 2024. The company highlighted the submission of its alfapump PMA to the US FDA, strong data from its DSR heart failure program, and plans for future clinical trials and commercialization.

The U.K. released detailed data on clinical trial sponsors failing to register trials, a first for any country. The Health Research Authority disclosed info on 1,545 trials, showing 92% were registered in 2022, with most sponsors informing the HRA of registration. Two-thirds were on ClinicalTrials.gov.

Stoke Therapeutics reported positive data from STK-001 studies in Dravet syndrome patients, showing significant seizure reduction and cognitive improvements. The FDA approved a dosing regimen, and the company plans regulatory discussions for a registrational study. STOK stock surged following the update.

Syndax Pharmaceuticals announces FDA Priority Review for revumenib, a menin inhibitor for relapsed/refractory KMT2Ar acute leukemia, with a PDUFA target action date of September 26, 2024, under the FDA's Real-Time Oncology Review Program.

IntraBio's NDA for IB1001, a treatment for Niemann-Pick Disease Type C, has been accepted by the FDA with a PDUFA date of September 24, 2024. IB1001 showed improvement in neurological signs, functioning, and quality of life in Phase 3 and IIb trials. IntraBio raised $40 million to support IB1001's commercialization.