Celgene Corp and Agios Pharmaceuticals Inc’s oral treatment for acute myeloid leukemia (AML) patients with a rare genetic mutation, has on Tuesday been approved by the U.S. Food and Drug Administration (FDA).
AML is a cancer that originates in the bone marrow and progresses rapidly, resulting in an abnormal increase in white blood cells. It is generally diagnosed in older people, and is uncommon before the age of 45. About 21,380 new cases of AML will be diagnosed in 2017 and some 10,590 patients will succumb to the disease this year, according to estimates by the American Cancer Society.
The drug, Idhifa, secured approval for relapsed or refractory AML patients who have an IDH2 mutation, the FDA said. The drug will have a monthly list price of $24,872, Celgene says noting that the median time on therapy for patients was 4.3 months in the trial to secure the FDA’s approval. The list price of a drug is not necessarily what patients actually pay. Their ‘out-of-pocket’ cost is based on their individual healthcare insurance plans and duration of treatment.
“While the product isn’t expected to be a big needle mover for Celgene, it does represent the first approval for a partnered
product,” J.P. Morgan analyst Cory Kasimov said, pointing out that the drug targeted “an area of clear high unmet need”.The drug is the first and only FDA-approved therapy for patients with an IDH2 mutation, a group that accounts for 8 to 19 percent of all AML patients. In the United States, that translates to about 1,200 to 1,500 patients, Celgene said.
The FDA gave Idhifa its orphan drug designation, as well as its Priority Review designation, under which the agency commits to taking action on an application within six months where the FDA determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing, or preventing a serious condition. The orphan drug designation “will grant (Celgene) greater Idhifa pricing power,” Dr. Newman added.
Common side effects associated with Idhifa include nausea, vomiting, diarrhea, increased levels of bilirubin, and decreased appetite. Women who are pregnant or breastfeeding should not take Idhifa because it may cause harm to a developing fetus or a newborn baby, the FDA said.
Idhifa’s approval comes with a boxed warning — the strongest mandated by the FDA — designed to call attention to the risk of differentiation syndrome, an adverse reaction that could be fatal if untreated.
Tuesday’s nod pushed the FDA’s new drug approval tally to 27 for the year after green lights for Gilead’s Vosevi, Puma’s Nerlynx and Janssen’s Tremfya last month. The agency approved just 22 new drugs last year.
