Europe orphan drugs

Milne, C., K. Kaitin, and E. Ronchi. 2004. Orphan Drug Laws in Europe and the US Incentives for the Research and Development of Medicines for Diseases of Poverty. WHO Commission on Macroeconomics and Health, CMH Working Paper Series, Paper No.WG2 9. Available at http //www.cmhealth.org/docs/wg2 paper9.pdf [Accessed May 10, 2005]. 

For the indications of PAH and chronic thromboembohc pulmonary hypertension, mondoBlOTECH received from the European Commission and the United States FDA the orphan dmg status designation. When reaching regulatory approval, this orphan drug status guarantees mondoBlOTECH a significant market exclusivity for the drug within Europe and USA. 

There are now several millions of American patients who have benefited from the Orphan Drug Act (Haffner 2002). There is every indication that the same will shortly be true in Europe and the rest of the world. 

Cystic fibrosis (CF) is a genetic disease caused by mutations to the gene coding the cystic fibrosis transmembrane conductance regulator (CFTR) protein in many epithelial cells and blood cells. It is the most common autosomal recessive disorder in Caucasians and affects approximately 28,000 patients in the United States and approximately 36,000 patients in Europe. An estimated 70,000 people worldwide have CF. The life expectancy of patients with CF has risen steadily over the last 25 years, from a median predicted age of survival of 25 years in 1985 to 37 years in 2011. Since in the United States an orphan disease is defined as an disease afflicting fewer than 100,000 patients, CF is an orphan disease, and ivacaftor (Kalydeco, 1) is an orphan drug. The drug is also known as a personalized medicine because CF affects about only 70,000 people worldwide. 

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