Orphan drug designation of MDPK67b for Netherton syndrome approved
Med Discovery has received a positive opinion from the EMEA on orphan medicinal product status for MDPK67b for the dermatology disorder Netherton Syndrome
The orphan drug designation status of MDPK67b for Netherton syndrome was approved by the COMP (Committee for Orphan Medicinal Products) of the EMEA. Netherton syndrome is an orphan genetic disorder leading to excessive skin shedding and other symptoms such as bamboo hair. It is particularly severe in babies and young children, where the impaired skin barrier function leads to dehydration and multiple opportunistic infections.
It is estimated that Netherton syndrome affects about 1 person in 200,000. At the root of this orphan disease is a mutation in a kallikrein inhibitor SPINK5, leading to increased kallikrein activity. There is currently no drug officially registered to treat Netherton syndrome. Emollients, keratolytics, and topical antibiotics remain the mainstays of symptomatic treatment. It is hoped that the kallikrein inhibitor MDPK67b applied topically will functionally replace the missing SPINK5 activity and give targeted symptomatic relief.
“We are very excited that the COMP has seen the potential of MDPK67b to help these suffering patients who currently have very few therapeutic options” said David Deperthes, CEO of Med Discovery. “We are working hard on the preclinical development of MDPK67b, hoping to be able to plan first clinical trials in Netherton syndrome in 2010”
For more information on Netherton Syndrome see: http://www.socialstyrelsen.se/english/international/rarediseases/nethertonsyndrome
For the COMP announcement, click on "more information" below. Note that Med Discovery was represented by Voisin consulting before the COMP.