Results for: Achondroplasia

BioMarin Pharmaceutical, a leading rare disease specialist, remains optimistic about its future despite recent competition from Ascendis Pharma. Despite Ascendis Pharma’s positive Phase 3 trial results for achondroplasia, BioMarin believes its Voxzogo drug will continue to perform well. This confidence is based on Voxzogo’s broad label without age restrictions, providing a competitive edge. BioMarin is targeting a significant revenue increase and improved operating margins, driven by Voxzogo’s growth and a new business structure. The company is also exploring external business development opportunities to further diversify its portfolio.

Ascendis Pharma’s TransCon CNP, a once-weekly treatment for achondroplasia, demonstrated superior annualized growth velocity compared to placebo in a recent trial. This could pose a significant challenge to BioMarin’s Voxzogo, the current leading treatment for achondroplasia. While BioMarin holds a first-mover advantage, the potential for TransCon CNP’s improved dosing schedule and safety profile could impact Voxzogo’s market share in the coming years.

Ascendis Pharma A/S (ASND) announced positive topline data from a Phase 3 trial of TransCon CNP (navepegritide) for children with achondroplasia, a genetic disorder causing dwarfism. The investigational drug demonstrated significant improvement in growth velocity compared to placebo, leading to a potential FDA application in the first quarter of 2025.