Chiesi Global Rare Diseases Announces FDA Approval of Ferriprox (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease


May 1, 2021

  • Approval is based on demonstrated reduction in liver iron concentration
  • Expanded indications for patients with sickle cell disease or other anemias, as well as thalassemia –

BOSTON, May 1, 2021 /PRNewswire/ — Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today that the U.S. Food & Drug Administration (FDA) has approved Ferriprox (deferiprone) for the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in adult and pediatric patients 3 years of age and older.* This FDA approval expands the use of Ferriprox for patients with SCD or other anemias as well as patients with thalassemia regardless of prior iron chelation exposure.
SCD affects about 100,000 people in the U.S. and leads to a lower life expectancy by more than 20 years compared to the general population. SCD patients are typically diagnosed prior to 2 years of age and the more severe patients start to experience pain crises early in childhood. Many require hospitalization and chronic blood transfusions to manage disease complications. Disease complications include early onset stroke, acute chest syndrome, and multiorgan failure. Renal complications affect nearly 30-50 percenti of patients with sickle cell anemia and account for 16-18 percent of mortality in adults with SCD.ii

“People who are living with SCD face significant challenges with pain and organ damage that can greatly impact their quality of life, and most who need blood transfusions also need iron chelation therapy including those with known kidney issues who have limited treatment options,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “We believe that delivering an iron chelation therapy that has no dosage adjustment required for patients with mild to severe renal impairment may address a significant unmet need in SCD. We have a long history of commitment to the rare disease community and this FDA approval is a testament to the investments we continue to make in scientific research and development with patients at the center of everything we do. We would like to thank the patients and the SCD community for their participation in clinical studies because without their support this would not have been possible.”

Ferriprox is a synthetic, orally active iron-chelating agent shown to be effective in reducing iron concentration by penetrating cell membranes and removing toxic iron from organ tissues and extracellular fluids. Ferriprox is available as a 1000mg twice-a-day oral tablet. A controlled study that compared the efficacy of Ferriprox to that of deferoxamine in patients with SCD and other transfusion-dependent anemias met the non-inferiority criterion for change in liver iron concentration from baseline after 12 months. Data from an extension study confirmed that liver iron concentration continued to decrease progressively over time with the mean value dropping from 14.93 mg/g dw at baseline to 12.30 mg/g dw after one year, to 11.19 mg/g dw after two years, and to 10.45 mg/g dw after three years of Ferriprox treatment. The most common adverse reactions reported during clinical trials in patients with SCD or other anemias were pyrexia, abdominal pain, bone pain, headache, vomiting, pain in extremity, sickle cell anemia with crisis, back pain, ALT increased, AST increased, arthralgia, oropharyngeal pain, nasopharyngitis, neutrophil count decreased, cough, and nausea.

To support patients treated with Ferriprox, Chiesi Global Rare Diseases offers the Chiesi Total CareSM Program. Through this program, healthcare professionals and patients can access a team of dedicated specialists able to provide individual support as well as information about prescription, reimbursement, financial assistance, and product refills. For information about Chiesi Total Care call 866-758-7071.

Indication and Important Safety Information


Ferriprox (deferiprone) is an iron chelator indicated for the treatment of transfusional iron overload due to:

  • thalassemia syndromes
  • sickle cell disease or other anemias

Ferriprox Tablets are indicated in adult and pediatric patients ≥8 years of age; Ferriprox Oral Solution is indicated in patients ≥3 years of age.

Limitations of Use

Safety and effectiveness have not been established for the treatment of transfusional iron overload in patients with myelodysplastic syndrome or in patients with Diamond Blackfan anemia.

Important Safety Information


  • Ferriprox can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis.
  • Measure the absolute neutrophil count (ANC) before starting Ferriprox and monitor weekly while on therapy.
  • Interrupt Ferriprox if infection develops and monitor the ANC more frequently.
  • Advise patients taking Ferriprox to report immediately any symptoms indicative of infection.

Ferriprox is contraindicated in patients with known hypersensitivity to deferiprone or to any of the excipients in the formulations.

In pooled clinical trials, 7.5% of 642 patients with thalassemia syndromes treated with Ferriprox developed increased ALT values. Four (0.62%) Ferriprox-treated subjects discontinued the drug due to increased serum ALT levels and 1 (0.16%) due to an increase in both ALT and AST. In pooled clinical trials, 7.7% of 196 patients with sickle cell disease or other anemias treated with Ferriprox developed increased ALT values. Monitor serum ALT values monthly during therapy with Ferriprox and consider interruption of therapy if there is a persistent increase in the serum transaminase levels. Decreased plasma zinc concentrations have been observed on deferiprone therapy. Monitor plasma zinc, and supplement in the event of a deficiency.

Ferriprox can cause fetal harm. Advise females of reproductive potential to use an effective method of contraception during treatment with Ferriprox and for at least six months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with Ferriprox and for at least three months after the last dose. Advise females not to breastfeed during treatment with Ferriprox and for at least 2 weeks after the last dose.

Avoid co-administration of Ferriprox with other drugs known to be associated with neutropenia or agranulocytosis; however, if this is unavoidable, closely monitor the absolute neutrophil count. Avoid co-administration with UGT1A6 inhibitors. Allow at least a 4-hour interval between administration of FERRIPROX and drugs or supplements containing polyvalent cations (e.g., iron, aluminum, or zinc).

The most common adverse reactions in patients with thalassemia (incidence ≥ 6%) are nausea, vomiting, abdominal pain, arthralgia, ALT increased and neutropenia. The most common adverse reactions in patients with sickle cell disease or other anemias (incidence ≥6%) are pyrexia, abdominal pain, bone pain, headache, vomiting, pain in extremity, sickle cell anemia with crisis, back pain, ALT increased, AST increased, arthralgia, oropharyngeal pain, nasopharyngitis, neutrophil count decreased, cough and nausea.

Inform patients that their urine might show a reddish/brown discoloration due to the excretion of the iron-deferiprone complex. This is a very common sign of the desired effect, and it is not harmful.

Advise patients to avoid alcohol while taking Ferriprox tablets (twice-a-day). Consumption of alcohol while taking Ferriprox tablets (twice-a-day) may result in more rapid release of deferiprone.

Please see full Prescribing Information, including boxed WARNING and Medication Guide.

About Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit

About Chiesi Group

Based in Parma, Italy, Chiesi is an international research-focused pharmaceuticals and healthcare group with over 85 years’ experience, operating in 30 countries with more than 6,000 employees (Chiesi Group). To achieve its mission of improving people’s quality of life by acting responsibly towards society and the environment, the Group researches, develops and markets innovative drugs in its three therapeutic areas: AIR (products and services that promote respiration, from new-born to adult populations), RARE (treatment for patients with rare and ultra-rare diseases) and CARE (products and services that support special care and consumer-facing self-care). The Group’s Research and Development centre is based in Parma and works alongside 6 other important research and development centres in France, the U.S., Canada, China, the UK, and Sweden to promote its pre-clinical, clinical, and regulatory programmes. Chiesi, since 2019, is the world’s largest B Corp certified pharmaceutical group. Chiesi Farmaceutici S.p.A. has changed in 2018 its legal status to a Benefit Corporation, by incorporating a double purpose for the creation of shared value, and to generate value for its business, for society and the environment. The global B Corp movement promotes business as a force for good. Moreover, as a Benefit Corporation, Chiesi Farmaceutici S.p.A. is required by law to include objectives of common benefit in its bylaws and to report annually in a transparent way. The Group is committed to becoming carbon neutral by the end of 2035.

  1. i Sundaram N, Bennett M, Wilhelm J et al. Biomarkers for early detection of sickle nephropathy. Am J Hematol 2011;86(7):559-66
    ii Nath KA, Hebbel RP. Sickle cell disease: renal manifestations and mechanisms. Nat Rev Nephrol 2015;11(3):1171-1171.

SOURCE Chiesi Global Rare Diseases

Related Articles:

Ferriprox (deferiprone) FDA Approval History

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