uniQure N.V. (NASDAQ: QURE) announced Wednesday September 2, 2026 the submission of a Biologics License Application (BLA) to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational AAV-delivered gene therapy for the treatment of Huntington's disease. The BLA is the first ever submitted for a Huntington's disease therapy of any kind. AMT-130 is delivered by stereotactic intraparenchymal injection into the striatum and expresses a microRNA designed to silence the mutant huntingtin (mHTT) gene. The submission is supported by three-year data from the Phase 1/2 program compared to a propensity score-matched external control from the Enroll-HD natural history database. uniQure also submitted a Marketing Authorisation Application (MAA) to the UK MHRA on the same day. A four-year data readout from the ongoing Phase 1/2 study is expected before end of Q3 2026. AMT-130 previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Approval would establish the first disease-modifying therapy for Huntington's, where standard-of-care remains symptom management with tetrabenazine and deutetrabenazine plus antipsychotics.
The FDA on Wednesday August 26, 2026 approved Rasonque (daraxonrasib, a once-daily oral pan-RAS inhibitor developed by Revolution Medicines) for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or are not candidates for multi-agent chemotherapy. Approval was based on the Phase 3 RASolute 302 trial across 500 previously-treated patients showing median overall survival of 13.2 months on Rasonque versus 6.7 months on standard chemotherapy. The drug does not require a companion diagnostic and is approved for patients with or without an identifiable RAS tumor mutation. FDA granted Breakthrough Therapy and Orphan Drug designations plus Priority Review. Daraxonrasib is a small molecule and not a peptide; the approval matters to peptide-relevant readers because it hardens the oncology backdrop the neoantigen peptide vaccine class (Merck-Moderna intismeran, individualized long-peptide programs) is entering, and because it validates a new mechanism class in a tumor where five-year survival has stalled near 13% for two decades.
Amylyx Pharmaceuticals on May 5 launched a U.S. Expanded Access Program (EAP) for up to 250 adults with post-bariatric hypoglycemia (PBH) following Roux-en-Y gastric bypass surgery. The investigational compound, avexitide, is a first-in-class peptide GLP-1 *receptor antagonist* — designed to bind GLP-1 receptors on pancreatic islet beta cells and block the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose. Avexitide carries FDA Breakthrough Therapy Designation for both PBH and congenital hyperinsulinism, plus Orphan Drug Designation. Topline data from the pivotal Phase 3 LUCIDITY trial are anticipated in Q3 2026. The program inverts the standard GLP-1 narrative — for this population, GLP-1 signaling is the problem.