The Food and Drug Administration on Friday approved the first drug in a new class of treatments for acute myeloid leukemia.
The drug — developed by Cambridge biotech Agios Pharmaceuticals — is a once-daily pill that might replace chemotherapy for some people with refractory or relapsing AML, a type of blood cancer that forms in the bone marrow.
About 700 to 1,100 people in the U.S. would be eligible for the drug under the label, approved for patients whose tumors have a mutation in a gene called IDH1. It will be marketed under the brand name Tibsovo.
“We really have seen only a very few drugs approved in the last 40 years for AML. It’s been a really hard nut to crack,” said Lee Greenberger, the chief scientific officer at the Leukemia and Lymphoma Society.
The drug, which is thought to block a tumor cell’s ability to divide and survive, comes with some serious potential side effects, including an irregular heart rhythm, along with milder side including fatigue, joint pain, and fever. The packaging will also include a boxed warning — indicating a potentially life-threatening risk. Tibsovo can cause a fatal reaction called differentiation syndrome, which causes organs to swell.
About 20 percent of the people who took the drug in one clinical trial developed the condition, which can be treated with steroids; all of them survived.)
Still, “the risk of differentiation syndrome is real,” said Dr. Courtney DiNardo, an oncologist at MD Anderson Cancer Center and the lead investigator of the drug’s early-stage clinical trial.
Agios and Celgene co-own Idhifa, another therapy for AML approved in August 2017 that targets a different mutation. Like Idhifa, Tibsovo received orphan drug status and doesn’t come cheap. The wholesale price for a 30-day supply is $26,115 — about the same as Idhifa’s price tag.
The average duration of therapy is four months, said David Schenkein, CEO of Agios.
Schenkein, CEO of Agios, said he was thrilled that the 10-year-old startup had won approval of its second drug, which came a month before the deadline for the FDA to act.
“It’s obviously an extremely exciting day for everybody here at Agios,” he said. “More importantly, it’s an exciting day for patients with acute myeloid leukemia who now have a treatment option that until today didn’t exist.”
Typically, people with refractory or relapsing AML who haven’t responded to other treatments will survive for less than six months, DiNardo said. Trials have not directly compared survival rates between people who received this treatment and people who did not. But data from one clinical trials showed that people who responded to Tibsovo survived for at least nine months on average. About 30 percent of people in the trial saw their disease go into complete remission; among these people, average survival was more than 19 months.
Doctors in the United States will be able to write prescriptions for the medicine starting Monday. According to its most recent annual report, the company plans to submit an application for approval to European regulators later this year.