Agios' first-in-class PK activator Pyrukynd scores FDA approval to treat rare blood disorder

After selling its cancer unit to Servier in 2020, Agios Pharmaceuticals has won the first FDA approval for its genetically defined diseases portfolio. With the approval for Pyrukynd to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency, the company is off and running in the commercial arena again.

Pyrukynd, an oral drug, on Thursday became the first FDA-approved medicine for the rare and debilitating blood disorder. The inherited disease causes lifelong complications including gallstones, pulmonary hypertension, osteoporosis and more. Agios notes it can cause patients to experience challenges at work and in their social lives.

Before the approval, the only options for treatment were red blood cell transfusions or spleen removal surgery, Agios says, which both come with risks and burdens.

The FDA based its approval on phase 3 studies called ACTIVATE and ACTIVATE-T. In the ACTIVATE study, the drug demonstrated a statistically significant increase in hemoglobin in patients who were not regularly transfused. In the ACTIVATE-T study, the drug reduced transfusion burden for those who were.

In a note to clients last month, Cantor Fitzgerald analyst Alethia Young wrote that her team expects 2022 sales of $21 million for the drug. For next year, the Cantor team expects sales of $73 million.

RELATED: Agios readies FDA filing as anemia drug clears another phase 3

Aside from PK deficiency, Agios is also testing the medicine in thalassemia and sickle cell disease. If the drug is able to score approval in those conditions, Pyrukynd could become a blockbuster, Agios CEO Jackie Fouse, Ph.D., told The Boston Globe.

"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," Fouse said in the approval statement.

Aside from Agios' commercial focus on its new launch, Cantor Fitzgerald's Young said her team thinks it's "fairly likely" the company will consider "making some strategic acquisitions over 2022" to expand its pipeline.

RELATED: Agios offloads oncology unit to Servier in $1.8B deal, zeroes in on genetically defined diseases

For the Cambridge, MA-based biotech, the approval comes after the $1.8 billion sale of its cancer unit to Servier in December 2020. Prior to the deal, the company's cancer meds Tibsovo and Idhifa had won FDA approvals.

Editor's note: This story was corrected to reflect Agios' prior presence in oncology.