BMN-331 is under clinical development by BioMarin Pharmaceutical and currently in Phase II for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency). According to GlobalData, Phase II drugs for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) have an 89% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how BMN-331’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
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.
BMN-331 overview
BMN-331 is under development for the treatment of hereditary angioedema (HAE). It comprises of adeno-associated virus 5 (AAV5) vector encoding SERPING1 gene. The therapeutic candidate acts by targeting hSERPING1. It is administered through intravenous route.
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 BMN-331’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
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.
