Orphan Europe has signed a licensing agreement with Rare Disease Therapeutics who will market Cystadane, indicated for homocystinuria, in USA and Canada.

Homocystinuria is a rare disease of genetic origin, affecting the methionine metabolism. This severe disease results in elevated homocysteine levels (toxic at these concentrations) throughout the body.

Homocystinuria causes a variety of sometimes severe symptoms involving mainly ocular, nervous and vascular systems, as well as the bones. These symptoms can include mental retardation, seizures, psychiatric disturbances, osteoporosis, scoliosis, marfanoid features and thromboembolic complications. The latter may lead to stroke, seizures, permanent neurologic sequellae and even death. If left untreated, 25% of homocystinuria patients die before the age of 30.

Cystadane® lowers the toxic homocysteine levels by activating an alternative metabolic pathway for the metabolization of homocysteine. Patients receiving a treatment effectively lowering homocysteine levels have a significantly reduced risk of potentially life threatening thromboembolic events.* Cystadane® has marketing approval in the US, Canada, Europe, Australia and Israel and is marketed directly by Orphan Europe in the rest of the world.

Rare Disease Therapeutics, Inc. (RDT) is a well established company founded in 1991 and located in Nashville, Tennessee, USA. RDT has a solid track record with multiple licensing agreements, a comprehensive global patient advocacy network, and significant success in drug development and approval. RDT works closely with the FDA Office of Orphan Product Development, National Organization for Rare Disorders, the National Institutes of Health, large international pharmaceutical companies, and patient advocacy groups to identify the unmet needs of patients with rare diseases and potential products to meet these needs.

“We are very pleased to be able to make Cystadane® available for homocystinuria patients in US and Canada” said Mr. Milton H. Ellis, President of RDT. “This acquisition reinforces our product portfolio of efficient treatment for rare disease patients.”

“In RDT we found our perfect partner for the important North-American market”, says Mr. William Gunnarsson, CEO of Orphan Europe.” They have the knowledge and commitment necessary to provide care for rare disease patients”.

* Yap S, Boers GH, Wilcken B, Wilcken DE, Brenton DP, Lee PJ,Walter JH, Howard PM, Naughten ER, Vascular outcome in patients with homocystinuria due to cystathionine beta-synthase deficiency treated chronically: a multicenter observational study, Arterioscler Thromb Vasc Biol. (2001 Dec) 21(12):2080-5. .

Source:  Orphan Europe website