long acting CNP (BMN 333) / BioMarin - LARVOL DELTA

Development of BMN 333, a long-acting version of vosoritide for FGFR3-related dwarfism and other skeletal dysplasias (ASBMR 2026) - No abstract available

Endocrine Disorders • Orthopedics • FGFR3

BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman Primates (ASBMR 2026) - No abstract available

Preclinical

BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman Primates (ASBMR 2026) - No abstract available

Preclinical

BMN 333 Achieves High Sustained Released Vosoritide Exposure with Favorable Safety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia (ENDO 2026) - "BMN 333 was well tolerated in adults, with no dose-limiting toxicities identified. Low Cmax and extended vosoritide exposure support the potential to safely improve growth with a weekly dosing schedule, which will be studied in phase 2/3 trials of BMN 333 in children with achondroplasia."

Clinical • First-in-human • P1 data • P2/3 data • Genetic Disorders • Orthopedics • FGFR3

333-301: A study of BMN 333 versus Vosoritide in Children with Achondroplasia (clinicaltrialsregister.eu) - P2/3 | N=25 | Not yet recruiting | Sponsor: Biomarin Pharmaceutical Inc.

New P2/3 trial • Genetic Disorders

ASPEN: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (clinicaltrials.gov) - P2/3 | N=160 | Recruiting | Sponsor: BioMarin Pharmaceutical | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders

ASPEN: A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (clinicaltrials.gov) - P2/3 | N=160 | Not yet recruiting | Sponsor: BioMarin Pharmaceutical

New P2/3 trial • Genetic Disorders