New Formulation Of Medication For Rare Disease Patients

Main Category: Blood / Hematology
Article Date: 07 May 2008 - 1:00 PDT

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A new treatment option - Ferriprox Oral Solution - is now available to patients suffering from thalassaemia major, a rare genetic disease with only 50 percent of the patients surviving the age of 35 years1. Thalassaemia patients require blood transfusions to survive, which leads to damaging iron accumulation in the tissues, especially the heart. Ferriprox (deferiprone) is an iron chelator that effectively removes the excess iron from the body caused by blood transfusions. A liquid formulation of Ferriprox is now available in the UK, Germany, Denmark, Norway and Sweden.

- For patients with Thalassaemia Major undergoing regular blood transfusions, removal of iron is essential. The introduction of an Oral Solution gives physicians and patients a greater choice of the treatments available. The new formulation of Ferriprox will make it easier for both existing and new patients to take their medication, said Dr Farrukh Shah, Consultant Haematologist, Whittington Hospital, London.

- In this rare disease, new treatment options are even rarer, said Bo Jesper Hansen, CEO, Swedish Orphan International, a distributor of Ferriprox in Europe. "The solution is ideal for patients who have difficulty swallowing tablets. It is our hope that this new treatment option will improve the compliance across the patient population and thus not only increase the quality of life for the rare disease patients in need but also reduce complications of the disease," he continued.

The traditional first-line treatment for thalassaemia-related iron overload is deferoxamine, but patients find the treatment difficult, since it involves subcutaneous infusion for eight hours, five or more nights per week. Furthermore, although deferoxamine prolongs life, about half of all patients still die before the age of 35, about 70% of them due to heart failure from iron accumulation in the myocardium1. Deferiprone was the first oral iron chelator available to thalassaemia patients. A key study now reports that deferiprone appears to be more effective than deferoxamine in removing iron from the heart 2,3, which may explain the reports of observational studies showing improved survival in patients with thalassaemia major4,5.

- Anything that makes it easier for a patient to take their chelation therapy is a great thing, and the announcement of this new formulation will help both physicians and patients alike; especially those who have difficulty with their current treatment, said Michael Michael, President UKTS, TIF Board Member.

About Thalassaemia

Thalassaemia is a rare, genetic disorder that affects the production of red blood cells. This leads to severe anaemia at an early age and patients require chronic blood transfusions to survive. Repeatedly transfusing a patient with blood eventually causes iron overload because of the iron in red blood cells. As there is no natural excretory mechanism for iron, it therefore accumulates in the body. If left untreated, iron overload can lead to severe damage of vital organs, particularly the heart.

About Ferriprox and Ferriprox Oral Solution

Ferriprox is indicated for the treatment of iron overload in patients with thalassaemia major when deferoxamine therapy is contraindicated or inadequate6. Ferriprox was first approved in the European Union in 1999. It has a well-established safety profile and has been used clinically in more than 7500 patients in more than 50 countries. Ferriprox oral solution is bioequivalent to Ferriprox tablets, shows the same benefit/risk profile and is also dosed three times daily.

Ferriprox has been developed by ApoPharma Inc., a part of the Apotex Group of companies. The company's expertise is focused mainly in the area of iron chelation.

References:

1. Survival in β-thalassaemia major in the UK: data from the UK Thalassaemia Register. The Lancet, Volume 355, Issue 9220, Pages 2051 - 2052 B. Modell, M. Khan, M. Darlison

2. Borgna-Pignatti C, Rugolotto S, De Stefano P, Zhao H, Cappellini MD, Del Vecchio GC, Romeo MA, Forni GL, Gamberini MR, Ghilardi R, Piga A, Cnaan A. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 2004;89(10):1187-1193.

3. Pennell DJ, Berdoukas V, Karagiorga M, Ladis V, Piga A, Aessopos A, Gotsis ED, Tanner MA, Smith GC, Westwood MA, Wonke B, Galanello R. Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood 2006;107(9):3738-3744.

4. Borgna-Pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, Ghilardi R, Piga A, Romeo MA, Rugolotto S, Zhao H, Cnaan A. Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood 2006;107 (9):3733-3737.

5. Telfer, P, Coen P, Christou S, Hadjigavriel M, Kolnakou A, Pangalou E, Pavlides N, Psiloines M, Simamonian A, Skordos G, Sitarou M, Angastiniotis M. Survival of medically treated thalassemia patients in Cyprus. Trends and risk factors over the period 1980-2004. Haematologica 2006; 91(9):1187-1192.

6. Ferriprox SPC Nov 2007.

Swedish Orphan International is specialized in the development, marketing and distribution of products used for the treatment of rare or life-threatening disorders, where current treatment is either unavailable or unsatisfactory. Swedish Orphan International's head office is in Stockholm, with wholly owned subsidiaries in all the Nordic countries and in England, Spain, Italy, France, Germany and the Czech Republic, plus representative offices in the three Baltic countries.

Swedish Orphan International