WSJ Reports: Small Biotech Gets Rights to Rare Disease Drug

January 7, 2015

The National Institutes of Health licensed rights to cyclodextrin to a biotechnology company that will take on development of the drug for the treatment of Niemann-Pick Type C disease.

The deal with the small, Gaithersburg, Md.-based, startup Vtesse Inc. is one of the first commercial efforts to come out of a program to develop drugs for rare and neglected diseases at the NIH’s National Center for Advancing Translational Sciences.

The NIH two years ago launched a clinical trial testing cyclodextrin’s safety in children with NPC disease, a fatal cholesterol metabolism disorder, following a discovery by scientists that mice and cats with NPC that got the drug appeared to live longer.

The cyclodextrin used in the NIH trial is made by Janssen Research & Development, a unit of Johnson & Johnson . Cyclodextrin has been used for years in the medical industry as an inactive substance to stabilize and dissolve drugs in solution.

Several years ago, a family with children suffering from NPC asked the Food and Drug Administration for permission to give cyclodextrin on an experimental basis to their children. Janssen later agreed to provide the drug free to families that got FDA permission and to the NIH, which launched a trial. Over the years, more families received FDA permission to use the drug under a doctor’s supervision.

Read more at The Wall Street Journal

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