The opinion will be subject to review by the EC, which finally grants the decision on orphan drug designation.
ATTR-Amyloidosis or TTR amyloidosis is a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
ISIS-TTRRx is an antisense drug being developed with GlaxoSmithKline (GSK), which has an option to exclusively license the ISIS-TTRRx program.
The drug is currently being evaluated in a Phase II/III trial in familial amyloid polyneuropathy (FAP) patients.
Isis chief operating officer Lynne Parshall said the company is encouraged by COMP’s continued recognition of the need for new treatments for rare and orphan diseases and the designation of its drugs as potential therapeutic options.
"Since the beginning of the year, we have received two positive opinions on European Orphan Drug Designation, first for ISIS-APOCIIIRx, our novel triglyceride-lowering drug, and now for ISIS-TTRRx," Parshall said.
"Our Phase 2/3 study for ISIS-TTRRx is underway with some patients treated for more than six months.
"FAP is a devastating life-threatening disease and patients with FAP have limited therapeutic options. We remain encouraged about the potential for ISIS-TTRRx to provide therapeutic benefit for these patients in need."
The COMP, a committee of the European Medicines Agency, adopts an opinion on the granting of orphan drug status, after which the opinion is submitted to the European Commission for endorsement of the opinion.
Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which accumulates in tissues, impairing their function.
In patients with transthyretin amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissues, including heart, peripheral nerves, and the gastrointestinal tract.