The FDA has approved Agios Pharmaceuticals Inc.’s Pyrukynd (mitapivat) for treating hemolytic anemia, a rare disease afflicting adults with pyruvate kinase (PK) deficiency.
The oral PK isozyme R activator is the first disease-modifying medication for treating PK deficiency, according to Cambridge, Mass.-based Agios.
The approval was announced on Feb. 17, which was the therapy’s PDUFA date, and Pyrukynd is expected to be available to patients in the U.S. within the next two weeks.
The price was not announced, though the company said it will offer access programs to reduce or eliminate patient out-of-pocket costs, including a program for lowering copays to $0 for commercially insured patients and an assistance program offering free prescriptions to eligible uninsured and underinsured patients.
Pyrukynd had a priority NDA that was granted in August along with the February PDUFA date. The company had submitted an MAA to the EMA in June.
The approval was based on two pivotal studies. Data from phase III studies showed 40% (n=16) of patients dosed in the Activate trial achieved hemoglobin response vs. 0 for placebo (two-sided p<0.0001), with average change from baseline of 1.67 g/dL for study drug vs. -0.15 g/dL for placebo (two-sided p<0.0001) at weeks 16, 20 and 24. Activate hit its primary endpoint.
In the Activate-T trial, 37% (n=10) of the participants achieved transfusion reduction response, which was defined as ≥33% reduction in transfusion burden in a 24-week fixed-dose period compared with individual historical transfusion burden (one-sided p=0.0002). Of these, nine achieved a ≥50% reduction. The Activate-T study also hit its primary endpoint in reducing the transfusion burden for patients who received transfusions.
Agios posted long-term extension data for treating PK deficiency with Pyrukynd at the American Society of Hematology annual meeting in December showing it maintained a hemoglobin response for up to 19.5 months and in an extension study for up to 21.9 months. Improved markers of ineffective erythropoiesis and iron metabolism were found in adults. The therapy was well-tolerated and had a consistent safety profile.
Nearly a year ago, at the beginning of April, the company closed the sale of its commercial, clinical and research-stage oncology portfolio to Servier Pharmaceuticals LLC. Agios received $1.8 billion cash up front for the portfolio and as much as $200 million in regulatory milestone payments for the investigational glioma treatment vorasidenib. Agios said the deal would help it accelerate and expand its genetically defined disease portfolio, including its work on Pyrukynd as a potential treatment for adults with pyruvate kinase deficiency, thalassemia and sickle cell disease.
Agios also has the drug in a phase II study for treating alpha-/beta-thalassemia. Data presented in June showed 16 of 20 (80%) patients met the primary endpoint, defined as ≥1 g/dL increase in hemoglobin concentration from baseline at one or more assessments between weeks four and 12 (one-sided p<0.0001), including all five with alpha-thalassemia and 11 of 15 with beta-thalassemia.