Dive Brief:

• The Food and Drug Administration has cleared a first-of-its-kind medicine Takeda Pharmaceutical and Protagonist Therapeutics developed for a rare blood disorder.
• Takeda said Friday that the agency approved rusfertide, which it will sell as Mimrylo for polycythemia vera, a cancer where excess blood cells build up and cause serious cardiovascular problems. Mimrylo is specifically cleared to treat the “erythrocytosis,” or abnormally high level of red blood cells, in adults with the condition. 
• According to Reuters, Mimrylo has a list price of $4,200 per vial. Notably, Mimrylo won a “broader-than-expected” label that doesn’t mandate prior treatment with other drugs such as Incyte’s Jakafi, wrote Jefferies analyst Stephen Barker. Those factors could support faster uptake and boost consensus revenue estimates, according to Barker. His team is projecting $2 billion in peak yearly sales.

Dive Insight:

Mimrylo is the first medicine of its kind for polycythemia vera. It mimics the activity of the liver hormone hepcidin, a mechanism that’s supposed to control iron levels and lower red blood cell production. Those effects are particularly important, according to Takeda, because despite standard treatments — like the drug hydroxyurea and phlebotomies — some 78% of patients still have an abnormally high percentage of red blood cells in their blood.

With 150,000 diagnosed patients in the U.S., Mimrylo “addresses a clear unmet need” by helping control red blood cell levels and reducing the need for phlebotomies, Barker wrote. 

In Phase 3 testing, around 77% of trial participants who received Mimrylo didn’t need phlebotomies over the 32-week evaluation period, compared to 32.9% of those in the placebo group. Injection site reactions and anemia were the most common adverse events reported. 

The treatment was originally discovered by Protagonist, which in 2024 licensed the therapy to Takeda for $300 million. Protagonist is receiving $275 million in payouts from Takeda related to Friday’s approval, and is eligible for another $875 million, plus sales royalties.

Mimrylo’s approval follows another regulatory green light earlier this month for Takeda’s narcolepsy drug Orzeyful and comes ahead of the expected clearance of a third medication important to the Japanese drugmaker’s future, the autoimmune treatment zasocitinib.

Takeda is looking to those three to help offset a decline in profits associated with patent losses. But Barker predicted that the company should “return to earnings growth next fiscal year.”