• BioMarin

    BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study

    Company Plans to Provide Topline Phase 3 Data by Year End Company Presents Data Confirming that the Phase 3 and Phase 2 Study Participants have Similar Baseline Parameters SAN RAFAEL, Calif., Nov. 14, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of disproportionate short stature in humans, at its annual R&D Day.  An ongoing, open-label, dose finding Phase 2 study of vosoritide for achondroplasia demonstrated over 54 months that children in cohort 3 (N=10) of the study, at a dose of 15 µg/kg/day, achieved a statistically significant (p< 0.005)…

  • Ascendis

    Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon CNP as Treatment for Achondroplasia

    COPENHAGEN, Denmark, Feb. 28, 2019 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technology to address significant unmet medical needs, today announced that the U.S. Food and Drug Administration(FDA) has granted Orphan Drug Designation (ODD) to TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Achondroplasia is the most common form of dwarfism and there are no FDA-approved therapies for this condition. Individuals living with achondroplasia may experience severe skeletal complications and comorbidities, including narrowing of the foramen magnum, sleep apnea and chronic ear infections. “We are pleased to receive Orphan Drug Designation for TransCon CNP in achondroplasia. Our TransCon CNP candidate…