uniQure Files Huntington's Gene Therapy — FDA Reversed Twice Already

uniQure filed AMT-130 for FDA and UK approval on three-year Phase 1/2 data with a propensity-matched external control, and a potential accelerated-approval action date in late April or early May 2027. The same day, Ultragenyx's Phase 3 Aspire study of apazunersen in Angelman syndrome missed both its primary and key secondary endpoints, and Akeso reported that ivonescimab met overall survival against pembrolizumab in first-line PD-L1-positive lung cancer. Also: Teva's anti-IL-15 antibody hits in a Phase 2a celiac study, Alteogen options its subcutaneous platform to Novartis for up to $3.223 billion, nine more manufacturers join the White House MFN pricing deals, and newly released memos show a CBER office director overruled her review team to approve Replimune's melanoma drug.Alex Mercer and Maya Patel host the Daily Roundup for September 2. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.