Clinical Trial News

Ascendis Pharma Announces Extension of U.S. Food and Drug Administration Review Period for TransCon PTH for Adults with Hypoparathyroidism

Yorvipath (palopegteriparatide) is FDA approved for hypoparathyroidism. Ascendis Pharma's review period for TransCon PTH extended to August 14, 2024.

Eton Pharmaceuticals Announces Submission to FDA of New Drug Application for ET-400 (Hydrocortisone Oral Solution)

Eton Pharmaceuticals submits New Drug Application to FDA for ET-400 (hydrocortisone oral solution), expecting a 10-month review for potential approval in Q1 2025.

Iterum Therapeutics Resubmits New Drug Application to U.S. Food and Drug Administration for Oral Sulopenem

Iterum Therapeutics resubmits NDA to FDA for oral sulopenem to treat uncomplicated urinary tract infections in adult women, with data from phase 3 clinical trials demonstrating its efficacy and safety.

Abeona Therapeutics Provides Regulatory Update on Pz-cel

Abeona Therapeutics receives a Complete Response Letter from the FDA for its Biologics License Application for prademagene zamikeracel (pz-cel) due to unresolved Chemistry Manufacturing and Controls (CMC) requirements, with no deficiencies identified in clinical efficacy or safety data. The company plans to resubmit the BLA in the third quarter of 2024.

Supernus Provides Regulatory Update for SPN-830

Supernus receives a Complete Response Letter from FDA for SPN-830, an apomorphine infusion device for Parkinson’s disease, citing product quality and infusion device master file issues. No clinical safety or efficacy issues identified. Company remains committed to addressing the CRL and resubmitting the NDA.

Stealth Biotherapeutics Announces FDA Acceptance of New Drug Application for Elamipretide for the Treatment of Barth Syndrome

Stealth BioTherapeutics announces FDA acceptance of New Drug Application for elamipretide, a potential treatment for Barth syndrome, supported by positive Phase 3 data. The FDA plans to hold an advisory committee meeting to discuss the application. Elamipretide has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations.

Jazz Pharmaceuticals Completes Zanidatamab Biologics License Application for Previously Treated HER2-Positive Metastatic Biliary Tract Cancer

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 Biologics License Application Accepted in the US for Patients with Previously Treated Metastatic HR-Positive, HER2-Negative Breast Cancer

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.

Milestone Pharmaceuticals Announces Resubmission of New Drug Application for Etripamil for Treatment in Paroxysmal Supraventricular Tachycardia

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 Announces Clinical Development Plan for Resubmission of New Drug Application for Reproxalap in Dry Eye Disease

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.
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