Masters Specialty Pharma, a UK headquartered pharmaceutical company specialising in emerging markets, and Addmedica, an independent French pharmaceutical company which develops and markets medicinal products for the treatment of serious rare diseases, have announced that they have signed an exclusive License and Distribution agreement for the sickle cell anaemia treatment Siklos® for Brazil.

Under the terms of the agreement, Masters will be responsible for the regulatory filing of the product with the Brazilian Health authorities and will be its exclusive distributor in the country.

Sickle Cell Disease is a serious, progressively debilitating and life-threatening genetic disease which manifests from birth. It affects the formation of red blood cells, which become unusually shaped and stick inside blood vessels, causing pain and depriving cells and organs of oxygen-carrying blood. The lifespan of the abnormal red cells in a sickle cell patient is around 100 times shorter than a healthy blood cell. This leaves patients’ blood chronically short of red blood cells resulting in severe anaemia.

“The registration of this in Brazil will ensure that the 60,000 children and adults with SCD have access to the treatment they need.”

Sickle Cell Disease affects millions throughout the world and is particularly common among certain ethnic groups. In Brazil, it represents a serious public health issue. Recent estimates suggest that approximately 2,500 children are born every year with sickle cell disease in the country, and there are many more carriers of the disease who lead normal lives but who have the potential to pass on the disease to their children.

Siklos®, which was developed by Addmedica, obtained European marketing authorisation in 2007 as the first and only medicinal product indicated in the prevention of recurrent painful vaso-occlusive crises in sickle cell anaemia. One of the advantages of Siklos® is that it offers greater dosage accuracy based on body weight in easy to administer 50mg increments.

Dr Zulf Masters OBE, founder and CEO of Masters Speciality Pharma said, “Siklos® is one of the first medicines that we will be made available to patients in Brazil through our business unit, Masters Specialty Medicines. Unlike the other treatments which are currently being used ’off-label’ to treat Sickle Cell Disease in Brazil, this medicine will be the first to be locally registered for the treatments of paediatric and adult patients with the disease.”

Dr Francois Anger, Chairman of Addmedica, added, “We are delighted that the long term relationship we have built with Masters over the last few years, has resulted in an agreement where our innovative product, Siklos®, can reach more patients, particularly in a part of the world where the disease is undertreated and novel therapies are needed.”

Clarisse Lobo, Clinical Research Specialist at HEMORIO, Medical Hematologist, MD in Clinical Sciences, Rio de Janeiro, Brasil, commented, “SCD is the most prevalent hereditary disease in Brazil, and its treatment is a public health priority. The registration of this innovative form of hydroxycarbamide 1,000mg and 100mg in Brazil will ensure that the 60,000 children and adults with SCD have access to the treatment they need.”

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