Earlier, Trappsol Cyclo was approved under compassionate use by the FDA in April 2009, to treat Addi and Cassi Hempel, identical twin girls suffering from NPC disease.
Reportedly, in March 2010, the Hempel family had filed an orphan drug application with the FDA for Trappsol Cyclo as the treatment for NPC. The family is also working with doctors to determine the most efficacious route of administration for the Trappsol Cyclo.
Rick Strattan, CEO of CTD Holdings, said: “There is no known cure for Niemann Pick Type C disease, nor is there any FDA approved treatment for this debilitating and relentlessly progressive condition. Trappsol Cyclo is showing promise in NPC afflicted experimental animals, and many international doctors treating NPC patients are expressing interest in the compound as a potential life extending therapy. Patients with the fatal condition have no other options currently.
“There is hope that cyclodextrin can not only help treat NPC disease, but there is solid evidence that other lysosomal storage diseases (LSD) can be treated.”