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Alnylam's TTR Amyloidosis Drug Receives Orphan Drug Designation in Europe

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Alnylam Pharmaceuticals said this week that its phase I transthyretin-mediated amyloidosis treatment ALN-TTR has received an orphan drug designation from the European Medicines Agency.

Specifically, the designation is for the use of the drug against familial amyloidotic polyneuropathy, one of the predominant forms of the disease, Alnylam said.

Orphan drug designation provides drug developers with financial incentives to develop therapies for diseases that affect small populations — no more than five in 10,000, according to EMA standards — as well as extended periods of market exclusivity.

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