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Isis' antisense drug ISIS-TTR Rx to receive European orphan drug status

Monday, February 17, 2014, 16:00 Hrs  [IST]

Isis Pharmaceuticals, Inc. has received the positive opinion from the Committee for Orphan Medicinal Products (COMP) recommending ISIS-TTRRx for designation as an orphan medicinal product for the treatment of ATTR-Amyloidosis to the European Commission (EC). The opinion will be subject to review by the EC, which ultimately 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 in development with GlaxoSmithKline (GSK), which has an option to exclusively license the ISIS-TTRRx programme. ISIS-TTRRx is currently being evaluated in a phase II/III study in familial amyloid polyneuropathy (FAP) patients.

"We are encouraged by COMP's continued recognition of the need for new treatments for rare and orphan diseases and the designation of our 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," said B. Lynne Parshall, chief operating officer at Isis. "Our phase II/III 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 designation, after which the opinion is submitted to the European Commission for endorsement of the opinion. Orphan drug designation is granted to products designed to diagnose, prevent or treat life-threatening or very serious conditions that affect not more than five in 10,000 persons in the European Union.

Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively 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. The presence of TTR aggregates interferes with the normal functions of these tissues, and as the TTR protein aggregates enlarge more tissue damage occurs and the disease worsens. The two most common types of TTR amyloidosis are familial amyloid cardiomyopathy, or FAC, which affects more than 40,000 patients worldwide, and familial amyloid polyneuropathy, or FAP, which affects more than 10,000 patients worldwide. ISIS-TTRRx is an investigational drug that is designed to inhibit the production of all forms of TTR, and could potentially offer an alternative approach to treat all types of transthyretin-related amyloidosis.

Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners.


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