Ultragenyx Pharmaceutical sold two priority review vouchers, each from an FDA approval of a gene therapy. Both therapies came from business deals and proceeds from the voucher sales put Ultragenyx in a better financial position to pursue yet another deal for a rare disease asset.
Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second approved gene therapy for Ultragenyx, which specializes in rare disease medicines.
In an interview, Kyan Health Co-Founder and Chief Commercial Officer Konstantin Struck discussed how Kyan gives mid-market and enterprise employers access to premium workforce mental healthcare, at a price point that is affordable.
Ultragenyx Pharmaceutical's apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The disappointing result has some readthrough to Ionis Pharmaceuticals and Oak Hill Bio, each in clinical development with Angelman drugs designed to work in a similar way as Ultragenyx’s failed molecule.
Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene therapy for Ultragenyx, which specializes in rare disease treatments.