Peptide News Digest

#Accelerated-Approval

2 stories

Regulatory · View digest

uniQure Submits BLA to FDA for AMT-130 (Ifezuntirgene Inilparvovec) AAV Gene Therapy for Huntington's Disease Under Accelerated Approval Pathway

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.

Regulatory · View digest

FDA Grants Accelerated Approval for Vera Therapeutics' Trutakna (Atacicept-Vymj) for Adult Patients with Primary IgA Nephropathy on Tuesday July 7: BAFF/APRIL-Targeting Fusion Protein Cut Proteinuria 45.7% Versus 6.8% for Standard of Care Alone at 36 Weeks in the ORIGIN Phase 3 Trial

The FDA granted accelerated approval Tuesday July 7, 2026 to Trutakna (atacicept-vymj) for adult patients with primary IgA nephropathy (IgAN) at risk of rapid disease progression, in combination with standard of care. Vera Therapeutics developed the drug as a recombinant fusion protein that combines the extracellular domain of the transmembrane activator and CAML interactor (TACI) receptor with the Fc portion of human IgG1, binding both B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL) to reduce autoantibody-driven kidney damage. The registrational Phase 3 ORIGIN trial data supporting approval: at 36 weeks, Trutakna plus standard of care produced a 45.7% reduction in urine protein-to-creatinine ratio versus a 6.8% reduction for standard of care alone. IgA nephropathy affects approximately 130,000 to 150,000 Americans and is the most common primary glomerular disease worldwide; approximately 40% of patients progress to end-stage renal disease within 20 years without adequate treatment. Trutakna is a fusion protein rather than a peptide but sits in adjacent therapeutic territory relevant to the site's peptide-and-biologic coverage. Vera Therapeutics (NASDAQ: VERA) is expected to launch the product in Q3 2026. Continued approval may be contingent on verification of clinical benefit in confirmatory Phase 3 studies.