ARTICLE | Politics & Policy

Clinicians protest EU Orphan Drug prices

November 24, 2010 1:10 AM UTC

In an open letter to U.K. Prime Minister David Cameron and Health Secretary Andrew Lansley, 20 neurologists and pediatricians called for a review of the price of drugs approved for Orphan indications. The letter cites the example of Orphan Drug Firdapse amifampridine, a formulation of 3,4-diaminopyridine marketed by BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) at an annual price of L40,000-L70,000 ($63,920-$111,860) for Lambert Eaton Myasthenic Syndrome (LEMS). The clinicians said the price of Firdapse is 50-70 times higher than the price of an unlicensed version of 3,4-diaminopyridine that has been used for more than 20 years to treat LEMS. The letter said that by receiving Orphan Drug status for the "slightly modified" version of 3,4-diaminopyridine, BioMarin was able to set Firdapse's price at "an exorbitant level with no clinically relevant advantage." The letter was published on Nov. 16 in the British Medical Journal. ...