Mitapivat sulfate is under clinical development by Agios Pharmaceuticals and currently in Phase III for Beta Thalassaemia. According to GlobalData, Phase III drugs for Beta Thalassaemia have an 83% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Mitapivat sulfate’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.

Mitapivat sulfate overview

Mitapivat sulfate (Pyrukynd) act as anti anemic agent. It is formulated as tablets, film coated tablets for oral route of administration. Pyrukynd is indicated for the treatment of hemolytic anemia (a disorder in which red blood cells are destroyed faster than they can be made) in adults with pyruvate kinase (PK) deficiency.

Mitapivat sulfate (AG-348) is under development for the treatment of hemolytic anemia in pediatrics with pyruvate kinase (PK) deficiency, alpha thalassemia and beta thalassemia and sickle cell disease. The drug candidate is a quinolone sulfonamide administered through oral route as a capsule and tablet. It targets pyruvate kinase receptor (PKR).

Agios Pharmaceuticals overview

Agios Pharmaceuticals (Agios) discovers and develops investigational medicines for the treatment of cancer and rare genetic metabolic disorders. Its pipeline products include AG-946, a novel, PKR activator for the treatment of hemolytic anemias and sickle cell disease. The company pipeline also includes mitapivat drug candidate to treat adult pyruvate kinase (PK) deficiency, adult thalassemia, adult sickle cell disease, pediatric pk deficiency, pediatric thalassemia, and pediatric sickle cell disease. Agios provides tablets under brand Pyrukynd for treatment of hemolytic anemia for adults with (PK) deficiency. The company offers myAgios patient support services program that includes access support, prescription fulfillment, financial assistance, disease education and community connections. Agios is headquartered in Cambridge, Massachusetts, the US.

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

This content was updated on 7 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.