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A drug trial is currently being conducted with OGT 918 (Zavesca or Miglustat) with NP-C patients. This compound proved to be very effective in NP-C mice in slowing the progression of the disease. Our hope is that it will do the same in humans.

Phase 1-2 of the trial is in progress with NP-C patients in the UK and the United States. There is also a pediatric trial.

The trial center in the US is at Columbia University under the direction of Marc Patterson, M.D. If you have questions on the trial, please contact Darlene Vecchio at Columbia University by phone at (212) 305-4136.

Important Information for Patients taking Zavesca

Miglustat animal toxicology data

Long term (two year) animal toxicity studies of miglustat have recently been reported. Mice given oral miglustat at doses of 210 (16 times the human dose), 420 and 840/500 mg/kg/day developed diarrhea, abdominal swelling and rectal prolapse. Microscopic examination of the large intestine showed inflammatory lesions. At the highest doses (840/500 mg/kg/day – 65/38 times the human dose) there was an increased number of tumors in the large intestine. Rats given the same doses of miglustat for two years did not develop such changes.

Male rats given miglustat in doses of 30, 60 and 180 mg/kg/day had an increased incidence of benign Leydig cell (testicular) tumors. These rats normally have a much higher incidence of Leydig cell tumors than humans. There was no increase in tumors in other organs.

We understand that many families will be concerned by these reports, and wish to place these in perspective:

• No cases of bowel or testicular tumors have been reported in humans taking miglustat (which has now been available in clinical trial and practice for many years)
• The doses of drug used in these animal studies are many times higher than those taken by humans.
• The toxic effects reported may be species specific, and not necessarily relevant to humans.
• Similar findings are not uncommon in toxicology studies.

We suggest that careful monitoring of gastrointestinal side effects such as is already performed, with further investigation for individuals who have persistent symptoms despite change in diet, dosage reduction or use of loperamide, is appropriate for individuals taking miglustat. You should speak to your physician directly to discuss any concerns that have not been addressed by this message.

Sincerely,

Marc C. Patterson, MD, FRACP
Professor and Director
Division of Pediatric Neurology

Dr. J.E Wraith (Consultant Paediatrician)
Willink Biochemical Genetics Unit
Royal Manchester Children's Hospital
Hospital Road, Manchester M27 4HA