Agios evaluating fourth Pyrukynd case after analyst report

Agios Pharmaceuticals (AGIO) disclosed in a regulatory filing that it is aware of the securities analyst report published today detailing recent Pyrukynd data from the Food and Drug Administration’s Adverse Event Reporting System received in connection with a Freedom of Information Act request. Agios added, “The Analyst Report references four patient cases. Three of the cases were reported to Agios, and subsequently reported to FDA, as part of the Company’s standard pharmacovigilance processes. Two of these cases relate to patients on commercially-available Pyrukynd, which is approved in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The third case relates to a patient with sickle cell disease who received treatment with Pyrukynd through individual patient expanded access, also referred to as compassionate use, at the request of the patient’s treating physician. Under FDA regulations and guidance, FDA permits such expanded access upon a determination that, among other things, the patient to be treated has a serious or immediately life-threatening disease or condition and there is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition. The fourth patient case was reported directly to the FDA. Agios is evaluating this case in connection with its pharmacovigilance processes…To date, the information available to the Company, including the cases referenced in the Analyst Report, has not altered the established benefit-risk profile of Pyrukynd which is represented in the USPI.” Shares of Agios are down 11% to $30.99 in afternoon trading.