Food and Drug Administration Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia

• If approved, 1st Therapy in U.S. for the Treatment of Achondroplasia
• PDUFA Action Date is Aug. 20, 2021

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. This acceptance by the FDA marks the first marketing application accepted for a treatment for achondroplasia in the United States.

The Prescription Drug User Fee Act (PDUFA) action date is Aug. 20, 2021. The FDA has informed the company that they are not currently planning to hold an advisory committee meeting to discuss the application.

Although the FDA did not identify any filing issues with the NDA, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. This information was included in the marketing application.

While not part of the vosoritide marketing application, in Q4, the Company is also expecting to complete enrollment in a Phase 2 randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia, aged zero to less than 60 months, for a period of 52 weeks. The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will complete a minimum three-month baseline study to determine their respective baseline growth prior to dosing in the Phase 2 study. Children in this study will have completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.

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