Agios Pharmaceuticals Scores FDA Approval For Tibsovo, Second AML Drug Win in Less Than a Year


FDA Approved

Shares of Agios Pharmaceuticals jumped this morning after the Cambridge, Mass.-based company said the U.S.Food and Drug Administration (FDA) approved its oral leukemia treatment Tibsovo (ivosidenib). It’s the second approval for the company in less than a year.

Tibsovo was approved for the treatment of patients with relapsed or refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation. This is the first and only medicine that the FDA has approved for this indication, Agios said this morning. Agios submitted its New Drug Application to the FDA in December and the FDA turned around a quick approval under Priority Review.

David Schenkain, Agios’ chief executive officer, called the approval an exciting milestone for the company. The FDA approval of Tibsovo is the company’s first wholly-owned drug to gain approval and the second medicine in its pipeline to be approved within a year. Last year Agios and its development partner Celgene won regulatory approval for Idhifa, a treatment for patients with relapsed or refractory AML who have an IDH2 mutation. Schenkain said Tibsovo will be geared toward the “6 to 10 percent of AML patients” with have an IDH1 mutation. Those patients have been waiting for a new treatment option that works “radically different than conventional chemotherapy.”

Acute myeloid leukemia is a cancer of the blood and bone marrow. It is marked by rapid disease progression and is the most common acute leukemia affecting adults. There are approximately 20,000 new cases estimated in the U.S. each year, Agios said. The majority of the AML patients typically relapse and they have a poor prognosis with a five-year survival rate of about 27 percent, the company said. For those 6 to 10 percent of AML patients with the IDH1 mutation, the enzyme blocks normal blood stem cell differentiation, which contributed to the genesis of acute leukemia, the company added.

Hagop M. Kantarjian, chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center, said IDH inhibitors represent a new class of noncytotoxic, targeted therapies for AML patients with IDH mutations.

Agios’ Tibsovo was approved based on positive Phase III data that showed patients on the drug achieved a combined complete remission (CR) and complete remission with partial hematologic improvement (CRh) rate. Trial data showed a CR+CRh rate of 32.8 percent. The CR rate was 24.7 percent (43 of 174 patients) and the CRh rate was 8 percent, the company said.

“AML patients who relapse or are refractory to available therapies have few, if any, treatment options,” Kantarjian said in a statement. “The clinical study demonstrated that Tibsovo has the potential to deliver strong, durable responses as a single agent and can help patients achieve and maintain transfusion independence.”

Shares of Agios hit a morning high of $93.71. 

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