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.