Ultragenyx Pharmaceutical

Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases.

Health Canada has extended approval of Evkeeza (evinacumab) to treat children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), a devastating inherited cholesterol disorder.

The FDA declined to approve Ultragenyx Pharmaceutical's experimental gene therapy UX111 for Sanfilippo syndrome, citing manufacturing and facility-related concerns rather than clinical efficacy issues.

The FDA issued a Complete Response Letter for Ultragenyx's UX111 AAV gene therapy, citing chemistry, manufacturing and controls (CMC) observations that the company believes are readily addressable.

Ultragenyx Pharmaceutical's Phase 3 Orbit study of setrusumab for osteogenesis imperfecta failed to meet the interim statistical threshold for early termination (p<0.01), leading to a 27% stock decline on July 6.

Ultragenyx and Mereo BioPharma announced that the Phase 3 Orbit study evaluating setrusumab (UX143) in pediatric and young adult patients with osteogenesis imperfecta is progressing toward final analysis around the end of 2025.

• The European Commission has approved Evkeeza (evinacumab) for treating children aged six months and older with homozygous familial hypercholesterolemia (HoFH). • Evkeeza is the first medicine approved in the EU for HoFH treatment in children as young as six months, addressing a critical unmet need. • Efficacy in young patients was predicted through model-based extrapolation, showing similar or greater LDL-C reduction compared to adults. • Evkeeza is now reimbursed and commercially available in multiple countries, with early access schemes in additional regions.

The FDA granted Breakthrough Therapy Designation to setrusumab for reducing fracture risk in osteogenesis imperfecta (OI) types I, III, and IV, for patients aged 2 years and older.

Ultragenyx's GTX-102, an investigational antisense oligonucleotide, demonstrates continued improvement across multiple domains in Angelman syndrome patients at Week 48 in Phase 1/2 data.

Ultragenyx presented positive Phase 1/2 data for GTX-102, an antisense oligonucleotide targeting Angelman syndrome, at the FAST Global Science Summit.