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Agios Pharmaceuticals files NDA for drug that would treat pyruvate kinase deficiency

By Brian Buntz | June 21, 2021

Agios The small-molecule therapeutics company Agios Pharmaceuticals (NSDQ:AGIO) has submitted a new drug application (NDA) with the FDA to use mitapivat to treat adults with pyruvate kinase (PK) deficiency, a rare condition in which the rapid breakdown of red blood cells occurs can trigger anemia. The condition is the result of an inherited lack of the enzyme PK.

Mitapivat is a novel oral, small molecule allosteric activator of wild-type and various mutated PKR enzymes.

“With this NDA filing, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong hemolytic anemia characterized by serious complications affecting multiple organs,” said Dr. Chris Bowden, the chief medical officer at Agios.

There are currently no FDA-approved therapies for PK deficiency. While mild cases may not require treatment, physicians treat more severe forms of the disease with blood transfusions and splenectomy. PK deficiency can result in a range of symptoms, including chronic fatigue, hemolytic crisis, iron overload, gallstones, splenomegaly, ulcers, liver cirrhosis, pulmonary hypertension and osteoporosis

The Cambridge, Mass.–based company filed the NDA based on ACTIVATE and ACTIVATE-T pivotal studies.

Agios Pharmaceuticals also plans to submit a marketing authorization application for mitapivat in the European Union in mid-2021.


Filed Under: clinical trials, Drug Discovery
Tagged With: Agios Pharmaceuticals, FDA, mitapivat, NDA, new drug application, pyruvate kinase
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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