Medical Need receives positive opinion on its application for orphan drug designation for the treatment of Wilson’s disease


Medical Need announced today that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency has issued a positive opinion for the company’s orphan drug designation application for choline tetrathiomolybdate for the treatment of Wilson’s disease. The positive opinion was issued at the COMP’s meeting on December 5-6, 2012 and has now been transferred to the European Commission for final decision.

“It is a great first step which has now been achieved in the regulatory path towards an EU approval for choline tetrathiomolybdate for the treatment of Wilson’s disease”, says Gudrun Hörnquist Director Regulatory and Quality Assurance at Medical Need, and continues; “The positive opinion by the COMP confirms the current unmet medical need among the Wilson’s disease patients and the medical plausibility of tetrathiomolybdate in this setting”

The final decision from the European Commission is expected in the beginning of 2013.


Wilson’s disease, also known as hepatolenticular degeneration, is an inborn error of metabolism resulting in a disturbed copper-metabolism. In affected patients, copper gradually accumulates in the body, including the liver and the central nervous system. The clinical presentation includes a broad range of symptoms, particularly common are liver impairment and neurological manifestations. The disorder typically presents in adolescence or early adulthood and requires life-long pharmaceutical intervention. If untreated, the disease is progressive and results in severe disability and death.


DecuprateTM is the proprietary bis-choline salt of tetrathiomolybdate, which presents superior stability and characteristics compared to other tetrathiomolybdate salts. Tetrathiomolybdate (TM) is a strong copper chelator with nano-molar affinity and does not bind any other physically relevant divalent cat-ion. TM has been tested in several clinical trials in Wilson’s disease (n=120) and was shown to rapidly bind and remove the excess copper, typically within weeks. In a comparative trial vs. standard of care in acutely presenting Wilson’s disease patients with neurological symptoms, TM has been shown to be significantly better at preserving neurological function. DecuprateTM has also been tested in oncological trials including approximately 150 patients treated up to three years, and was found to be safe and tolerable while efficiently lowering copper levels with once daily dosing. Key side effects primarily relate to over-excessive copper-depletion, resulting in bone marrow suppression, and were rapidly reversible with a decrease of the daily dose. DecuprateTM is owned and developed on a global basis by Wilson Therapeutics AB, and Medical Need holds the exclusive rights to the product in Europe.


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.


Medical Need is a privately held pharmaceutical company headquartered in Sweden, focusing on the registration, distribution, marketing and sale of orphan drugs and niche specialty pharmaceuticals across Europe for the treatment of rare diseases where there is a high unmet medical need. More information on Medical Need can be found on