Agios Reports the US FDA’s Acceptance of NDA and Priority Review for Mitapivat to Treat Pyruvate Kinase Deficiency

August 18, 2021 Auto Bot Acceptance, Agios, FDA, NDA, Priority Review, regulatory 0

Shots:

  • The NDA & MAA submissions are based on ongoing 2 studies i.e., ACTIVATE & ACTIVATE-T evaluates safety, tolerability & efficacy of mitapivat vs PBO in patients with PKD
  • Results: 40% of patients achieved a hemoglobin response, 33% reduction in transfusion burden @ 24wks. fixed dose period & safety profile was consistent with previously reported data. The full results including PROs were presented at EHA 2021
  • The company has also submitted an MAA to EMA for the mitapivat in Jun’21 & has launched the myAgios patient support services program for patients & caregivers with PKD. Additionally, US FDA has granted a PR designation of mitapivat’s NDA with a PDUFA date is Feb 17, 2022

Click here to­ read full press release/ article | Ref: Globe Newswire | Image: Agios

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