Last week on July 25th Alnylam announced the Health Canada approval of Onpattro (patisiran) for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated (hATTR) amyloidosis. Onpattro is the first RNA interference (RNAi) therapeutic approved in Canada.
hATTR is a rare condition, found in 50 000 people worldwide, caused by an inherited mutation of the TTR gene. This causes abnormal amyloid protein to accumulate in the body and cause debilitating symptoms such as: peripheral sensory-motor neuropathy, autonomic neuropathy, cardiomyopathy, and death.
Messenger RNA (mRNA) transcribes genetic information from DNA and carries it to ribosomes, which translate the mRNA into protein. Onpattro targets and degrades TTR mRNA, preventing the translation of mRNA into abnormal amyloid protein.
Alnylam has one other RNAi therapy in registration phase and five others in late stage. We’re looking forward to seeing how this breakthrough technology can help patients in Canada with hATTR and other diseases!