Vosoritide is under clinical development by BioMarin Pharmaceutical and currently in Phase III for Dwarfism. According to GlobalData, Phase III drugs for Dwarfism does not have sufficient historical data to build an indication benchmark PTSR for Phase III. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Vosoritide LoA Report. Buy the report here.
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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Vosoritide overview
Vosoretide (Voxzogo) is a modified recombinant human C-type natriuretic peptide acts as bone growth regulator. It is formulated as powder for solution and solution for subcutaneous route of administration. Voxzogo is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed., which occurs after puberty when children reach final adult height and Idiopathic Short Stature. Voxzogo is indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.
Vosoritide (BMN-111) is under development for the treatment of achondroplasia, hypochondroplasia, Turner syndrome, SHOX deficiency, osteogenesis imperfecta, Noonan syndrome and genetic causes of short stature (dwarfism). The drug candidate is formulated as a lyophilizate solution for injection administered through subcutaneous route. It is an analog of C-type natriuretic peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth. It acts by targeting atrial natriuretic peptide receptor 2 (NPR2).
BioMarin Pharmaceutical overview
BioMarin Pharmaceutical (BioMarin) is a biotechnology company that develops and commercializes innovative medicines for the treatment of rare genetic diseases and medical conditions such as Duchenne muscular dystrophy (DMD), achondroplasia, phenylketonuria (PKU), late infantile neuronal ceroid lipofuscinosis (CLN2) and hemophilia A. The company focuses on developing breakthrough treatments for debilitating and life-threatening diseases. Its product category consists of multiple clinical and preclinical offerings which include Naglazyme-Mucopolysaccharidosis Type VI (MPS VI), Aldurazyme-MPS I, and others. The company operates through its manufacturing facility in Novato, California. It conducts business operations in the US, Europe, Latin America, the Middle East, and Asia-Pacific. BioMarin is headquartered in San Rafael, California, the US.
For a complete picture of Vosoritide’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
