The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from 10 months to six months from the day of filing acceptance. The FDA’s Priority Review designation is given to investigational medicines that treat a serious condition and offer significant improvements in safety or effectiveness.
“The acceptance of our NDA for Mitapivat with priority review represents an important milestone on the path to expeditiously 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 Sarah Gheuens, M.D., Ph.D., Senior Vice President of Clinical Development and Incoming Chief Medical Officer at Agios. “We look forward to working with the FDA during the review process and will continue to execute on our global strategy to ensure we are well-positioned to rapidly deliver Mitapivat to patients and healthcare providers upon approval.”
Agios also submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) in June 2021 for Mitapivat as a potential treatment for adults with PK deficiency. As announced on the company’s second-quarter 2021 earnings call, the MAA passed validation which triggered the start of the MAA review procedure.
The NDA and MAA submissions are based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. A full analysis of these data – including patient-reported outcomes (PRO) – was recently presented at the European Hematology Association (EHA) Virtual Congress. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability, and efficacy of treatment with Mitapivat.
Mitapivat has also been granted Orphan Drug Designation for the treatment of patients with non-transfusion-dependent α- and β-thalassaemia (NTDT). In addition to the Phase 2 extension study of The experimental drug in adults with NTDT α- and β-thalassemia, Agios is initiating two Phase 3 studies of Mitapivat in adults with thalassaemia in the second half of 2021.