New EU orphan drug designation for the treatment of Congenital Sucrase-Isomaltase Deficiency
Medical Need Europe AB earlier announced that the European Commission has approved the drug designation for Sucraid® for the treatment of Congenital Sucrase-Isomaltase Deficiency (CSID) in the EU. The EC decision followed the positive opinion adopted by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency at its meeting in July 2013. For further information we today publish the public summary of opinion on the orphan designation. PublicSummaryOpinion2013-9-16
“The orphan drug designation confirms the severely debilitating nature of CSID and the current unmet medical need for these patients in Europe”, says Gudrun Hörnquist, Director Regulatory and Quality Assurance at Medical Need Europe AB, and continues; “We are delighted to collaborate with our US partner QOL Medical towards a regulatory approval of Sucraid (sacrosidase), to improve availability of the product, which is currently provided on a named patient basis in most European countries . In parallel to the regulatory process, we collaborate with academia and genetic laboratories to develop and make available a simple genetic test to increase the precision and ease of making the diagnosis which we believe will further improve the situation for patients and health care. ”
The orphan drug designation was filed by QOL Therapeutics EU Ltd, in collaboration with Medical Need.
ABOUT CONGENITAL SUCRASE-ISOMALTASE DEFFICIENCY
Congenital sucrase-isomaltase deficiency (CSID) is a rare inherited disorder caused by malfunction of an enzyme in the gut called sucrase-isomaltase, responsible for the degradation and uptake of certain sugars in the food. Sucrase-isomaltase is the primary enzyme for degradation of dietary starch and sucrose, together responsible for over 90% of carbohydrates in the Western diet. In the absence of a functioning sucrase-isomaltase enzyme, these sugars are not absorbed and remain in the gut, resulting in chronic malnutrition and severe and chronic gastrointestinal symptoms, such as frequent watery diarrhea, excessive gas, abdominal pain and cramps, sometimes accompanied by vomiting. Children with CSID often fail to thrive and fall behind in their expected growth and development curves. CSID-patients have the condition from birth, but symptoms present as the children are introduced to normal food and are exposed to sugar. However, due to the similarity of symptoms with several other conditions, such as irritable bowel syndrome (IBS), patients may often remain undiagnosed up into adulthood and then frequently report a long history of severe and disabling gastrointestinal problems, often accompanied by a significant healthcare consumption. The abundance and wide-spread presence of sucrose in foods makes it very hard to successfully avoid exposure through dietary management. The severe symptoms of CSID may affect the patient’s ability to attend school or work in a normal way, and they may as a consequence not be able to develop their full potential with regards to education, profession, and social life.
Sucraid® (sacrosidase) is an enzyme replacement therapy for CSID, containing an enzyme with high capacity for degrading sucrose. Taken with food, it substitutes the cardinal dysfunction of CSID, and replenishes the body’s capability to absorb dietary sucrose, significantly alleviating the symptoms. Sucraid® is approved as an orphan drug in the US since 1998, and is currently used across Europe on a so called named-patient basis. Medical Need holds the distribution rights to the product in Europe, including Turkey.
About Orphan drug designation
The EU orphan drug legislation was adopted in 2000 with the goal of stimulating development of pharmaceuticals for rare conditions with unmet medical needs, so called orphan drugs. A rare condition is defined as a condition affecting less than 5 out of 10 000 individuals across the EU. Under the legislation, companies developing such pharmaceuticals may apply for an orphan drug designation of their product, which makes it eligible for several incentives, including free assistance, fee reductions and waivers throughout the development and, following marketing authorization, ten years market exclusivity.