Evinacumab is under clinical development by Ultragenyx Pharmaceutical and currently in Phase II for Heterozygous familial hypercholesterolemia (heFH). According to GlobalData, Phase II drugs for Heterozygous familial hypercholesterolemia (heFH) have an 88% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Evinacumab’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.

Evinacumab overview

Evinacumab (Evkeeza) is a fully human monoclonal antibody, acts as anti-hypercholesterolemia. It is formulated as injectable solution, and concentrate solution for intravenous route of administration. Evkeeza is indicated as an adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH).

Evinacumab (REGN-1500) is under development for the treatment of dyslipidemia in homozygous familial hypercholesterolemia (HoFH), heterozygous familial hypercholesterolemia. The drug candidate is administered through subcutaneous and intravenous route. It targets angiopoietin-like 3. The therapeutic candidate is a monoclonal antibody developed based on the VelocImmune technology.

It was also under development for the treatment of hypertriglyceridemia and acute pancreatitis.

Ultragenyx Pharmaceutical overview

Ultragenyx Pharmaceutical (Ultragenyx) is a biotechnology company that develops novel medicines for rare and ultra-rare genetic diseases. It has four marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH); Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

For a complete picture of Evinacumab’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 20 February 2024

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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.