LONG ACTING DEFERIPRONEΤο FDA και το
BOSTON, May 21, 2020 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate1. The new formulation of twice-a-day Ferriprox 1000 mg oral tablets eliminates the mid-day dose.
“The availability of a new twice-a-day oral tablet formulation of Ferriprox provides patients with the ability to reduce serum ferritin levels and cardiac and liver iron,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases “This important milestone for Chiesi Global Rare Diseases is another reflection of our commitment to innovation to improve the lives of patients living with rare diseases around the world.”
Thalassemia syndromes are a group of rare inherited hematological conditions including beta-thalassemia and are characterized by impaired hemoglobin production. In certain cases, such as with beta-thalassemia, severe forms of this genetic disorder if left untreated can lead to life-threatening complications. Patients are often managed with long-term blood transfusions that can put them at risk of developing very high levels of iron in their blood and vital organs. As the level of iron rises, it can generate free radicals that can be toxic to proteins and membranes. Deferiprone is engineered to bind to iron in the tissues and circulation, thereby inactivating it. Iron is then excreted from the body primarily via urine. Dosing is initiated at 75 mg/kg/day and can be increased up to 99 mg/kg/day to improve efficacy in iron chelation. The application for marketing approval was supported by bioequivalence studies.
“In the management of patients with thalassemia, clinicians often see firsthand the difficulties they can have with dosing and compliance, and the impact that this can have on the effectiveness of treatment,” said Thomas Coates, M.D. Section Head, Hematology at Children’s Hospital Los Angeles. “A treatment option that reduces serum ferritin, cardiac iron and liver iron with an established safety profile and now twice-a-day tablet dosing can represent a significant advantage for patients.”
To support patients treated with Ferriprox (twice-a-day), Chiesi Global Rare Diseases offers the Chiesi Total Care™ Program. Through this program, HCPs 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.
Ferriprox (twice-a-day) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.
Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival.
TIF ‘s HIGH-LEVEL THALASSΑEMIA& SICKLE CELL DISEASE HEALTHCARE PROFESSIONALS SUMMIT
THESSALONIKI 1-2 NOVEMBER 2019
Vasili Berdoukas is a haematologist working in Greece, in private practice.
He was a Visiting Professor in Pediatrics at the University of Southern California in the Division of Hematology/Oncology and Bone and Marrow Transplantation, Children’s Hospital of Los Angeles, USA. The University of Southern California between 2009 and 2014 and has been awarded an Emeritus Professorship in Pediatrics from the USC.
He completed his undergraduate medical degree at the University of Sydney in 1971 and his pediatric training in Sydney, London and Athens. He also completed a law degree at the University of New South Wales graduating in 2001.
He has worked in the field of haemoglobin disorders for almost 40 years. In London, in 1976-1977 he worked with Professor Bernadette Modell, then in 1978-79 in Greece with Professor Dimitris Loukopoulos, where he established the service of prenatal diagnosis of thalassaemia and other significant haemoglobin disorders.
From 1981 – 2000 he was a Staff Paediatrician at the Sydney Children’s Hospital with a particular interest in haematology and particularly thalassaemia. During that time he also established prentatal diagnosis for haemoglobinopathies there as well. Until he went to the United States in 2009 he worked at the “Aghia Sophia”Children’s Hospital in two of the largest Thalassemia units in Greece.
He was co-author in a book on the Clinical Approach to Thalassaemia. He has at least 50 publications in the field of paediatrics and haematology.