Mimrylo

Mimrylo is used to treat erythrocytosis (high red blood cell count) in adults with polycythemia vera. It is given by subcutaneous (under-the-skin) injection once or twice weekly.

Mimrylo (rusfertide) works by mimicking hepcidin, the body's own iron-regulating hormone. Like hepcidin, it blocks ferroportin, the transporter that releases iron from cells into the bloodstream. Less circulating iron means less is available for red blood cell production, which lowers hematocrit. Mimrylo belongs to the drug class called hepcidin mimetics, and is the first in its class to be approved.

Mimrylo (rusfertide) received FDA approval on August 28, 2026. FDA approval was supported by the global, randomized Phase 3 VERIFY study (NCT05210790), in which Mimrylo met all efficacy endpoints.

• The primary endpoint was the proportion of patients responding during Weeks 20–32, defined as not needing to withdraw blood (an absence of phlebotomy eligibility - confirmed hematocrit ≥45% at least 3 percentage points above baseline, or a hematocrit ≥48%).
• Key secondary endpoints at Week 32 were the mean number of phlebotomies, proportion maintaining hematocrit <45%, and change in fatigue (PROMIS Fatigue Short Form 8a) and total symptom burden (MFSAF v4.0).

Before using Mimrylo, tell your healthcare provider about all of your medical conditions, including if you:

• are pregnant or plan to become pregnant. Mimrylo may harm your unborn baby. Females who can become pregnant: Your healthcare provider may do a pregnancy test before you start treatment with Mimrylo. Use effective birth control (contraception) during treatment and for at least 30 days after the final dose of Mimrylo. Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with Mimrylo. Stop taking Mimrylo if you become pregnant.
• are breastfeeding or plan to breastfeed. It is not known if Mimrylo passes into your breast milk. Do not breastfeed during treatment with Mimrylo and for 30 days after the final dose of Mimrylo. Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

It is not known if Mimrylo is safe and effective in children.

Serious side effects

Mimrylo may cause serious side effects, including:

• New or worsening increased blood platelets (thrombocytosis). Mimrylo may increase blood platelet counts. Your healthcare provider will do blood tests when you start and during treatment with Mimrylo to check your platelet counts.
• Injection-site reactions. Mimrylo may cause injection-site reactions such as redness, itching, pain, swelling, bruising, hardening at the injection site, and irritation. If you get an injection site reaction, apply ice packs, steroid cream, or take medicines as needed to treat injection site pain and swelling.
• Harm to an unborn baby. Based on animal data, Mimrylo can cause harm to an unborn baby. Females who can have children should use effective contraception. See "Before Using" below.

Your healthcare provider may change your dose or stop treatment with Mimrylo if you have side effects.

Common side effects

The most common side effects of Mimrylo are:

• injection site reactions
• low red blood cell count (anemia).

These are not all of the possible side effects of Mimrylo.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

See the detailed Instructions for Use that come with Mimrylo for information on how to prepare and inject a dose of Mimrylo.

• Use Mimrylo exactly as your healthcare provider tells you to.
• Your healthcare provider will tell you how much Mimrylo to inject and when to inject it. Do not inject more than your prescribed dose.
• Do not change your dose or stop treatment with Mimrylo unless your healthcare provider tells you.
• Mimrylo is given as an injection under your skin (subcutaneous injection).
• Inject Mimrylo under the skin in the stomach area (abdomen) at least 2 inches away from the belly button (navel), front of the thigh, or the upper outer arm. Choose a different site each time you inject Mimrylo.
• Your healthcare provider should show you or your caregiver how to prepare and inject Mimrylo. Contact your healthcare provider if you have questions. Do not inject yourself or someone else until you have been shown how to inject Mimrylo.
• Mimrylo is available in more than 1 strength. Make sure the Mimrylo strength you have matches your prescribed strength.
• Your prescribed dose may require more than 1 injection. Do not give more than two injections a day. If your weekly dose is greater than 82 mg, give your first injection on day 1 and the second injection on day 4 or 5.

• If you inject one time a week: If you miss a dose by 1 to 4 days, take the missed injection as soon as you remember. Then continue with your regular weekly schedule. If you miss a dose by more than 4 days, take the missed injection as soon as you remember. Take your next injection 3 days later and then return to your regular weekly schedule.
• If you inject two times a week: If you miss a dose by 1 to 2 days, take the missed injection as soon as you remember. Take your next injection 3 days later and then return to your regular schedule. If you miss a dose by more than 2 days, skip the dose and continue with your regular injection schedule.
• Contact your healthcare provider if you do not know when to inject a missed dose.

• Store Mimrylo at room temperature between 68°F and 77°F (20°C and 25°C).
• Do not freeze.
• Store Mimrylo in the original carton to protect it from light.
• Do not use the vial or diluent syringe after the expiration date on the carton.
• After mixing (reconstitution), Mimrylo may be stored at room temperature between 68°F and 77°F (20°C to 25°C) for up to 4 hours.
• Use Mimrylo within 4 hours after mixing.
• Throw away (dispose of) mixed Mimrylo if not used within 4 hours.

Keep Mimrylo and all medicines out of the reach of children.

Active ingredient: rusfertide acetate.

Inactive ingredients: mannitol, sodium acetate trihydrate, and glacial acetic acid/sodium hydroxide for pH adjustment.

The diluent for Mimrylo contains sodium acetate trihydrate, zinc acetate dihydrate, and glacial acetic acid.

Mimrylo is distributed by Takeda Pharmaceuticals America, Inc., Cambridge, MA 02142

For more information, go to www.MIMRYLO.com or call 1-877-TAKEDA-7 (1-877-825-3327).