The Food and Drug Administration on Thursday approved a new drug from Agios Pharmaceuticals to treat people with a rare form of anemia.
The Agios drug, mitapivat, is the first approved treatment for pyruvate kinase deficiency, a rare, inherited disorder that results in the rapid destruction of red blood cells. Given as a pill, mitapivat is designed to activate an enzyme called pyruvate kinase-R that red blood cells use to convert sugars into energy.
PK deficiency affects approximately 3,000 people in the U.S.
The drug will be marketed under the brand name Pyrukynd at an annual price of $334,880, before insurer’s discounts.
Pyrukynd is the first new drug approval for Agios since it sold its cancer business, including two approved drugs, to the French pharmaceutical company Servier in December 2020. Agios now focuses on developing treatment for rare diseases.
“For more than a decade, we have been pioneering the science of PK activation in order to bring Pyrukynd to people with PK deficiency and provide them with the first medication approved specifically to address this rare, debilitating blood disorder,” Jackie Fouse, CEO of Agios, said in a statement.
The FDA approved Pyrukynd based on two Phase 3 clinical trials that demonstrated the drug’s ability to increase hemoglobin and decrease the need for blood transfusions. Commonly reported side effects included decreased reproductive hormones in males, back pain, joint swelling, and increased blood lipids.
European regulators are reviewing Pyrukynd for PK deficiency, with a decision expected by the end of 2022. Agios is conducting additional studies to expand the use of the drug to include children with PK deficiency and other diseases affecting red blood cells, including thalassemia and sickle cell disease.