Agios Pharmaceuticals, Inc, a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, announced that the US health regulator has approved PYRUKYND (mitapivat) in the US for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia. PYRUKYND is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease.

“The successful ACTIVATE and ACTIVATE-T studies demonstrate the impact of mitapivat in significantly improving hemolysis and anemia in PK deficiency,” said Hanny Al-Samkari, MD, hematologist and clinical investigator at the Mass General Cancer Center and Harvard Medical School, and an investigator in these pivotal Phase 3 studies. “The US FDA approval of mitapivat, a targeted agent and first disease-modifying medication in PK deficiency, is an encouraging step forward for these patients that addresses a significant unmet need.”

“PYRUKYND is the first approved therapy for PK deficiency and marks an important milestone for these patients, who may face tremendous challenges and debilitating symptoms throughout the course of this lifelong disease,” said Rachael Grace, MD, MMSc, pediatric hematologist, director of hematology clinical research at Boston Children’s Hospital and an investigator in the Phase 2 DRIVE PK and Phase 3 ACTIVATE studies. “Partnering with Agios and the PK deficiency community to improve understanding of the natural history of this rare disease and bring a new medicine to patients has been an honor, and I look forward to additional collaboration in the future.”

“I am so grateful that PYRUKYND has been approved for PK deficiency. As both patient and caregiver, I spent the majority of my life feeling alone in this disease and never thought I would see a medicine approved,” said Kim Hall, who was diagnosed with PK deficiency in 1969 and is the mother of two adult daughters living with PK deficiency. All three women participated in the Phase 3 PYRUKYND PK deficiency clinical program. “The experience of being part of the clinical trials has been impactful because of the connections we have built with other patients, healthcare providers and Agios colleagues who understand PK deficiency and are actively working to improve patients’ lives.”

“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,” said Jackie Fouse, CEO at Agios. “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. These connections have fueled today’s tremendous milestone for the PK deficiency community. Each of us at Agios is dedicated to making a difference for people with PK deficiency, as well as to expanding the reach of PYRUKYND and our clinical and research programs to many more patients with genetically defined diseases around the world.”

PYRUKYND is expected to be available in the US approximately two weeks after approval. PYRUKYND was reviewed by the FDA under Priority Review and was previously granted orphan drug designation. PYRUKYND is also under review by the European Medicines Agency as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU by the end of 2022.

The FDA granted approval to PYRUKYND based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.