Ferriprox (deferiprone)
An approved treatment for Aceruloplasminemia.
The same compound appears under different names depending on the context. Here is how to identify Deferiprone wherever you encounter it, plus the key facts at a glance.
- Generic name
- Deferiprone
- Brand name
- Ferriprox
- Development code
- L1
- Drug class
- Iron chelator
- Manufacturer
- Chiesi
- How it's taken
- Deferiprone is taken by mouth.
An iron chelator that can reach iron deposits in the brain, which is especially relevant for aceruloplasminemia since the disease causes iron accumulation in brain tissue. Its FDA label covers iron overload from blood transfusions in thalassemia, sickle cell disease and other anemias, not aceruloplasminemia, so its use here is off-label.
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Where Deferiprone fits
Alternative or add-on iron chelator, often used when deferasirox is insufficient or not tolerated. Sometimes combined with other chelators for patients with severe iron overload.
How Deferiprone works
Deferiprone binds to excess iron in your blood, forming a complex that your body can eliminate through urine. Unlike deferasirox, deferiprone can cross the blood-brain barrier, meaning it can reach iron deposits in the brain where they cause damage in aceruloplasminemia.
Mechanism: Oral iron chelator with ability to cross the blood-brain barrier
Side effects and safety
The most serious potential side effect is agranulocytosis (dangerously low white blood cell count), which is a boxed warning on the label. In clinical trials it occurred in about 1% of patients with thalassemia and 0.5% of patients with sickle cell disease or other anemias. Other common side effects include nausea, vomiting, abdominal pain, joint pain, and raised liver enzymes. Regular blood tests are essential. Report fever or signs of infection immediately. The label also warns that deferiprone can harm an unborn baby, so women who could become pregnant should use effective birth control during treatment and for at least 6 months after the last dose, and men with partners who could become pregnant should do so for at least 3 months after.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Deferiprone
Deferiprone is taken by mouth. Ferriprox tablets come in a twice-a-day version and a 3-times-a-day version, and there is also an oral solution. It has a shorter action time than deferasirox, so it is taken more than once a day. Requires careful monitoring with frequent blood tests.
Availability and cost
Generic versions may be available at a lower cost. Ask your pharmacist.
Iron chelator available in generic form. Small molecule oral medication used when patients need an alternative or additional chelation approach.
Help paying for Ferriprox
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Ferriprox Copay Program: eligible commercially insured patients may pay as little as $0; up to $10,000 per calendar year. Government-insured patients excluded.
For: private insurance · source - Insurance and case manager help
Patient service coordinators, reimbursement specialists, pharmacists and nursing support help with insurance and refills.
The official page does not say who qualifies. Ask the program. · source
Good to know: Covers brand-name Ferriprox only; generic deferiprone is available. Copay program not available in California or Massachusetts when an AB-rated generic exists. Uninsured patients may qualify for more help, but details are not given.
Clinical trial results
FDA approved deferiprone on October 14, 2011, for iron overload from transfusions. Research suggests it may be helpful for preventing iron-related brain damage in conditions like aceruloplasminemia where iron accumulates in brain tissue.
Development history
Deferiprone was developed internationally and approved in several countries before FDA approval in the U.S. in 2011. It offers an alternative to deferasirox with the unique advantage of crossing the blood-brain barrier.
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Common questions about Deferiprone
▸What is Deferiprone (Ferriprox)?
An iron chelator that can reach iron deposits in the brain, which is especially relevant for aceruloplasminemia since the disease causes iron accumulation in brain tissue. Its FDA label covers iron overload from blood transfusions in thalassemia, sickle cell disease and other anemias, not aceruloplasminemia, so its use here is off-label.
▸How does Deferiprone work?
Deferiprone binds to excess iron in your blood, forming a complex that your body can eliminate through urine. Unlike deferasirox, deferiprone can cross the blood-brain barrier, meaning it can reach iron deposits in the brain where they cause damage in aceruloplasminemia.
▸What are the side effects of Deferiprone?
The most serious potential side effect is agranulocytosis (dangerously low white blood cell count), which is a boxed warning on the label. In clinical trials it occurred in about 1% of patients with thalassemia and 0.5% of patients with sickle cell disease or other anemias. Other common side effects include nausea, vomiting, abdominal pain, joint pain, and raised liver enzymes. Regular blood tests are essential. Report fever or signs of infection immediately. The label also warns that deferiprone can harm an unborn baby, so women who could become pregnant should use effective birth control during treatment and for at least 6 months after the last dose, and men with partners who could become pregnant should do so for at least 3 months after.
▸How is Deferiprone taken?
Deferiprone is taken by mouth. Ferriprox tablets come in a twice-a-day version and a 3-times-a-day version, and there is also an oral solution. It has a shorter action time than deferasirox, so it is taken more than once a day. Requires careful monitoring with frequent blood tests.
▸Is Deferiprone FDA approved?
Yes, Deferiprone (Ferriprox) is FDA approved (2011) for the treatment of Aceruloplasminemia.
▸Why might deferiprone be preferred for aceruloplasminemia?
Deferiprone can cross the blood-brain barrier more effectively than deferasirox, which may be important for removing iron that has accumulated in the brain in aceruloplasminemia. Brain iron deposition is a major cause of neurological symptoms in this condition.
▸What is the most serious side effect of deferiprone?
Agranulocytosis (severe drop in white blood cells) is the most serious risk, which can be life-threatening due to infection susceptibility. The label calls for a neutrophil count before starting, then weekly for the first 6 months, every 2 weeks for the next 6 months, and every 2 to 4 weeks after the first year.
▸How is deferiprone taken?
Deferiprone (Ferriprox) is taken by mouth either 2 or 3 times a day, depending on which tablet is prescribed. More than one dose a day can be harder to keep up with than once-daily deferasirox.
▸Is deferiprone specifically approved for aceruloplasminemia?
Deferiprone (Ferriprox) tablets are approved for iron overload from blood transfusions in people 8 and older with thalassemia, sickle cell disease, or other anemias. Its use in aceruloplasminemia is based on clinical experience with its iron chelation properties, particularly its ability to cross the blood-brain barrier.
▸What monitoring is required?
Blood count (neutrophil) monitoring for agranulocytosis is the most important requirement: weekly for the first 6 months, every 2 weeks for the next 6 months, then every 2 to 4 weeks. Liver enzymes (ALT) are checked monthly and zinc levels yearly.