The U.S. Food and Drug Administration has approved Agios Pharmaceuticals’ candidate treatment for a rare type of hemolytic anemia.
The regulator gave PYRUKYND (mitapivat) the green light for adults diagnosed with pyruvate kinase (PK) deficiency, an inherited disease that starts as chronic hemolytic anemia characterized by rapid red blood cell destruction. The inherited mutation in the PKLR gene may cause huge deficits in red blood cells and can lead to serious complications, including osteoporosis, gallstones, extramedullary hematopoiesis, pulmonary hypertension and iron overload. People with PK deficiency experience poor quality of life. Treatments are available but they come with short-term and long-term risks.
Pyrukynd is the first approved disease-modifying treatment for this disease.
The FDA’s decision is based on positive results from Agios’ Phase III ACTIVATE and ACTIVATE-T trials, demonstrating the drug’s ability to improve hemolysis and anemia issues in PK deficiency.
In the ACTIVATE study, 40% of participants reported improved hemoglobin response (the primary endpoint) compared to zero patients in the placebo group. The trial also achieved its secondary endpoints of ineffective erythropoiesis and markers of hemolysis. In the ACTIVATE-T study, 33% of the patients achieved a transfusion reduction response at the end of 24 weeks, while 22% were transfusion-free. Adverse reactions were the same in both trials, most of which were mild and short-term.
An extension trial on patients from both studies is ongoing to evaluate Pyrukynd’s long-term tolerability, safety and efficacy of treatment. Agios said that it plans to launch a similar study by mid-2022 but this time for pediatric patients with PK deficiency who are regularly transfused and not regularly transfused. These will be named ACTIVATE-kids and ACTIVATE-kidsT.
The company is also advancing its Phase III ENERGIZE and ENERGIZE-T trials in adults diagnosed with thalassemia and another Phase II/III RISE UP study in patients who have sickle cell disease.
“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. We remain committed to partnering with patients, caregivers, advocates and healthcare providers to ensure that the impact of PYRUKYND® is maximized through robust support, education and access programs,” commented Jackie Fouse, Ph.D., chief executive officer at Agios, in a statement.
Agios is rolling out programs that will make Pyrukynd easily accessible to all, including a co-pay system that lowers copays to $0 for eligible commercially-insured patients. It also offers a Patient Assistance Program that provides prescriptions for free.Pyrukynd is expected to be available to adults in the U.S. two weeks after the FDA approval. The drug was previously granted Orphan Drug Designation and was evaluated under Priority Review. Agios also submitted an application with the European Medicines Agency for adults with PK deficiency and expects feedback by the end of 2022.