Teijin Pharma Limited has announced that Japan's Ministry of Health, Labor and Welfare $(MHLW.SI)$ has granted Orphan Drug designation to navepegritide for the treatment of achondroplasia, a rare genetic skeletal dysplasia. Developed as TransCon CNP and licensed from Ascendis Pharma, A/S, navepegritide is designed for use in children with achondroplasia in Japan. The drug, which is administered subcutaneously once per week, utilizes TransCon technology to prolong C-type natriuretic peptide $(CNP)$ action. Teijin Pharma plans to initiate a Phase III clinical trial of navepegritide domestically in the first half of fiscal year 2025. Ascendis Pharma has filed a new drug application in the United States and plans to submit a Marketing Authorization application in Europe later in 2025. Achondroplasia affects approximately one in every 20,000 live births and is considered an intractable disease by the MHLW.
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