Clinical Trial News

The FACHILD study focuses on evaluating the modified Friedreich Ataxia Rating Scale (mFARS) in pediatric patients with Friedreich's ataxia (FRDA), a rare hereditary condition. The study highlights the rapid disease progression in children, the challenges in assessing disease status using mFARS, and the need for pediatric trials to explore therapeutic interventions. It also discusses the impact of the COVID-19 pandemic on data collection and the importance of FARS E (Upright Stability) as a predictor of disease progression.

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.

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.

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.

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.