Pharmaprojects R&D Pipeline News Feed
First hyperphenylalaninaemia drug filed in Europe
23 Nov 2007
Merck Serono has filed a marketing approval application with the EMEA for its hyperphenylalaninaemia (HPA) drug sapropterin. As there are currently no approved products for the treatment of HPA, sapropterin could be of potential benefit to tens of thousands of HPA patients.
There are estimated to be 50,000 patients in the developed world who suffer from HPA as a result of a genetic disorder which disables them from metabolizing the common amino acid phenylalanine, causing it to reach toxic levels in the blood. Currently, patients’ only option is diet restriction in order to limit the amount of phenylalanine ingested, a course which can prove hard to maintain. Sapropterin is estimated to be of use in 30-50% of these patients.
Sapropterin is the synthetic form of 6R-BH4, a naturally-occurring cofactor to phenylalanine hydroxylase, the enzyme which normally metabolizes phenylalanine. In two Phase III trials in 179 HPA patients, sapropterin significantly reduced blood phenylalanine levels and allowed patients to tolerate an increased intake of phenylalanine.
Sapropterin is currently only available in Japan, although Merck Serono’s development partner BioMarin filed for US approval in May, where it has received orphan drug and fast-track status.
