The antisense drug ISIS-TTR, a part of the Isis-GSK strategic RNA therapeutic alliance, is being developed for the treatment of transthyretin (TTR) amyloidosis.
TTR amyloidosis is a severe and rare genetic disease that causes progressive dysfunction of peripheral nerve and/or heart tissues.
Isis chief operating officer and chief financial officer Lynne Parshall said ISIS-TTR was granted orphan drug status and fast track designation for FAP patients.
"ISIS-TTR is our most advanced drug from our severe and rare disease franchise and represents a significant near-term commercial opportunity for us," Parshall added.
"We look forward to continuing to move ISIS-TTR toward the market for patients who have very limited therapeutic options."