ACHONDROPLASIA TREATMENT MARKET ANALYSIS

Market News

Global Achondroplasia Treatment Market: Key Developments

On March 7, 2023, BioMarin Pharmaceutical Inc., a biotechnology company dedicated to transforming lives through genetic discovery, announced that the U.S. Food and Drug Administration (FDA) had accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) for injection to expand treatment in the U.S. to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of disproportionate short stature.

In August 2020, Ascendis Pharma A/S, a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, announced that the European Commission (EC) had granted Orphan Designation to TransCon C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of dwarfism. TransCon CNP is an investigational long-acting prodrug of CNP designed to provide the continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. TransCon CNP also received in 2020, orphan designation for the treatment of achondroplasia in the U.S.

On June 1, 2023, Myriad Genetics, Inc., a company of genetic testing and precision medicine, launched new studies and expansion of its Precise Oncology Solutions portfolio at the 2023 American Society of Clinical Oncology (ASCO).

In January 2020, Wacker Biotech is technological leader in the chemical industry and manufactures products for all key global industries. The company Provides Active Ingredient to treat achondroplasia in children global phase-2 Trial for Ascendis Pharma is a biopharmaceutical company that focuses on developing drugs for endocrinology and oncology indications.