ANVISA also approves FERRIPROX in combination with other iron chelators in thalassemia major when monotherapy is ineffective
BOSTON, Mass. – September 13, 2021 - Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today that Brazil’s National Health Surveillance Agency (ANVISA) has approved FERRIPROX® (deferiprone) for the treatment of iron overload in patients with sickle cell disease (SCD) or other anemias.
The company also announced ANVISA has approved FERRIPROX in combination with another chelator in patients with thalassemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload justifies rapid or intensive correction. FERRIPROX was previously approved in Brazil as a monotherapy for the treatment of iron overload in patients with thalassemia major when current chelation therapy is not recommended.
“We are pleased to announce these new indications for FERRIPROX in Brazil in sickle cell disease and as a combination therapy in thalassemia major, which marks the first time in 15 years that an iron chelation therapy has been approved in the country,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “This is an important moment for patients living in Brazil who face significant challenges with damage due to iron overload, as well as their doctors who now have an expanded arsenal of treatment options for these rare and underserved conditions.”
“This approval is a meaningful achievement that will benefit a significant portion of patients affected by sickle cell disease, which is the most common hereditary anemia in Brazil,” said Carlos Chueiri, Medical Director of Chiesi Brazil. “The approval of FERRIPROX in sickle cell disease confirms Chiesi's commitment to innovation as we are always seeking to meet the broadest possible needs of patients and caregivers.”
Thalassemia major and sickle cell disease are two disorders that affect red blood cells. Both conditions cause problems with hemoglobin, depriving many parts of the body of oxygen. People living with these conditions often require chronic blood transfusions that can put them at risk of developing very high levels of iron in their blood and vital organs. 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.
U.S. Food & Drug Administration Approved Indication and Important Safety Information
FERRIPROX® (deferiprone) is an iron chelator indicated for the treatment of transfusional iron overload due to:
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
WARNING: AGRANULOCYTOSIS AND NEUTROPENIA
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 is contraindicated in patients with known hypersensitivity to deferiprone or to any of the excipients in the formulations.
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.
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 https://www.chiesiglobalrarediseases.com/.
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.
For further information: www.chiesi.com.
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