Ultragenyx FAYUVI Sanfilippo Type A First-Ever FDA Approval, Novo Rebrand, Dualitas + Roche $1B Bispecific Deal

The FDA granted standard full approval to Ultragenyx's FAYUVI (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy that the company calls the first approved treatment for Sanfilippo syndrome type A; treated children scored 23.5 points higher on cognitive testing than natural-history controls, and BioSpace reported a $3.95 million list price. Dualitas Therapeutics signed a bispecific antibody discovery deal with Roche worth up to $1 billion, including $36.5 million upfront, to screen more than 300,000 combinations for immunology and inflammation targets. NICE reversed its earlier rejection of Enhertu for HER2-low advanced breast cancer, citing a commercial agreement, higher cost-effectiveness thresholds, and a new method for assessing quality of life. Novo Nordisk's Monday move to 'Novo' as its day-to-day name set up its September 21 Capital Markets Day, and the comment period for the FDA's 17 revised draft guidances on generic peptides, including semaglutide and tirzepatide, closes on September 28.

Correction (September 23, 2026): This digest was revised to remove claims that could not be traced to a primary source and to replace general source links with specific ones. An item describing a September 17 BMO Capital Markets note on retatrutide was removed because the note and the TRIUMPH-2 and TRIUMPH-3 results it discussed were reported in July 2026. The FDA's peptide guidances were published on July 28, 2026, not in early September as first stated. Unsupported DESTINY-Breast04 figures, an after-hours Ultragenyx share price, Enhertu approval dates, and Indian generic market estimates were also removed.