Takeda’s blood cancer drug Mymlyro wins FDA approval as ‘first in its class’

The U.S. Food and Drug Administration (FDA) has approved Takeda’s blood cancer treatment Mymlyro (luspertaide) for polycythemia vera (PV).

According to the FDA and the news outlet Fierce Pharma on the 28th, Mymlyro is the first treatment to introduce a new mechanism of action for PV, a rare blood cancer in which the bone marrow produces too many red blood cells. PV can cause life-threatening cardiovascular complications, including stroke and myocardial infarction.

Mymlyro is the first PV treatment to mimic hepcidin, a hormone that naturally regulates iron in the body. It works by limiting the iron released into the bloodstream, thereby suppressing excessive red blood cell production.

Existing first-line treatments include phlebotomy (blood removal), hydroxyurea and interferon to keep hematocrit below the safe threshold of 45%. Incyte’s JAK inhibitor Jakafi is used as a second-line treatment option.

Dr. Tanya Roblewski of the FDA’s Center for Drug Evaluation and Research said, “Patients with PV have had to contend with the consequences of excess red blood cell production through frequent blood draws. This approval provides a first-in-class option in a new class that can meaningfully reduce the burden on patients.”

According to Takeda, about 90,000 people in the U.S. have PV, and about 78% of them are unable to control their hematocrit with existing standard treatments.

Mymlyro demonstrated efficacy in the Phase 3 VERIFY study. Among 293 patients with uncontrolled hematocrit who received weekly subcutaneous injections for 32 weeks, the proportion who improved to a level at which phlebotomy was not needed during weeks 20-32 was 76.9% in the Mymlyro group and 32.9% in the placebo group.

Patients treated with Mymlyro also had fewer phlebotomies and a statistically significant improvement in fatigue. The most common adverse reactions were injection-site reactions and anemia.

This is Takeda’s second FDA drug approval since CEO Julie Kim officially took office in June. Earlier this month, the sleep disorder treatment Orzepul was approved for narcolepsy type 1.

Kim said, “Mymlyro’s approval demonstrates the strength of Takeda’s late-stage pipeline and our focus on developing differentiated treatments for patients awaiting new options.”

Takeda plans to draw on its existing expertise in blood cancers as it launches Mymlyro. The company has the multiple myeloma treatments Velcade and Ninlaro, as well as the leukemia treatment Iclusig.

Mymlyro was originally developed by Protagonist Therapeutics. Takeda secured certain rights in 2024 with an upfront payment of $300 million, then acquired its partner earlier this year. Jefferies analysts estimated Mymlyro’s peak sales at about $2 billion.