Clinical Trial News

The U.S. FDA has approved the expanded use of TRIKAFTA for treating cystic fibrosis in patients aged 2 and older with specific CFTR gene mutations, making approximately 300 more people eligible for this treatment. The approval includes updates to safety information regarding liver injury and liver failure.

PTC Therapeutics, Inc. has announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, supported by promising clinical trial data.

Verastem Oncology has announced that the FDA has accepted its New Drug Application for avutometinib in combination with defactinib for the treatment of recurrent low-grade serous ovarian cancer with a KRAS mutation, granting it Priority Review with a PDUFA target action date of June 30, 2025.

FDA approved CARVYKTI® for treating relapsed/refractory multiple myeloma, reducing disease progression or death risk by 59% vs standard therapies. Based on Phase 3 CARTITUDE-4 study, it's the first BCMA-targeted therapy for early relapse, offering a potential treatment-free period.

Axsome Therapeutics has successfully completed its Phase 3 clinical trial, ACCORD-2, for Alzheimer’s disease agitation, achieving both primary and key secondary endpoints with significant results compared to placebo.

Stoke Therapeutics has achieved alignment with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the EMPEROR study, focusing on zorevunersen's effects on major motor seizure frequency, behavior, and cognition in children and adolescents.

Acute technical success was achieved in nearly all patients, with 96% showing no or trivial PR. 86% were discharged after one day; extended stays were due to chest pain, arrhythmia, or procedural issues. Follow-up showed mild or less PR in 98%, with high freedom from adverse outcomes. TPV-related thrombosis and endocarditis were rare but notable. Real-world outcomes were excellent, with ongoing follow-up planned.

Sanofi's cancer drug SAR444245, part of a $2.5B acquisition, returns to Phase 1/2 testing due to lower than expected efficacy, leading to a €1.6B impairment. The IL-2 based therapy aims for a safer, effective cancer treatment amidst competition from other biotechs innovating IL-2 therapies.

Jazz Pharmaceuticals completes BLA submission for zanidatamab, a HER2-targeted bispecific antibody, seeking accelerated approval for treatment of previously-treated HER2-positive metastatic biliary tract cancer. If approved, zanidatamab would be the first HER2-targeted treatment for this indication in the U.S. The BLA includes data from the Phase 2b HERIZON-BTC-01 trial showing a confirmed objective response rate of 41.3% in HER2-positive BTC patients, with a manageable safety profile.

Datopotamab deruxtecan's BLA accepted in the US for treating metastatic HR-positive, HER2-negative breast cancer, based on Phase III trial results showing significant PFS improvement over chemotherapy. FDA decision expected Q1 2025.