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Isis familial amyloid polyneuropathy candidate wins FDA fast track status

Isis Pharmaceuticals has obtained the US FDA fast track designation for ISIS-TTR to treat familial amyloid polyneuropathy (FAP).

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."