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news.Alnylam has received positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) regarding orphan drug status for ALN-TTR01, as a treatment for familial amyloidotic polyneuropathy (FAP).
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ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of all forms of ATTR, which includes FAP and familial amyloidotic cardiomyopathy (FAC).
Alnylam Regulatory Affairs vice president Saraswathy Nochur said they believe that RNAi therapeutics targeting transthyretin could represent a major advance for the treatment of ATTR – a significant unmet medical need where breakthrough therapies are needed.
"Our ongoing Phase I trial is actively enrolling patients and is focused on demonstrating safety and tolerability of ALN-TTR01; importantly, it is also designed to capture key data on clinical activity through measurements of serum TTR levels," Nochur said.
"We are on track to complete enrollment in this study by the end of the first half of this year and to report data in the third quarter."