At the BIO International Convention held in Philadelphia, PA, we talked with Akshay Vaishnaw, MD, PhD, Chief Medical Officer at Alnylam Pharmaceuticals about the company’s approved RNA interference (RNAi) drug, Patisiran, to treat to hereditary transthyretin-mediated amyloidosis (ATTR).

Dr. Vaishnaw said, “Hereditary ATTR is caused by a mutated gene that gives rise to a mutant TTR messenger RNA in the liver cell, that gives rise to mutant transthyretin protein that comes out of the liver cell, circulates in the blood, and deposits in the heart and the nerves. And when that happens usually by middle-aged people, it manifests a severe neuropathy that’s damage to the nerves and cardiomyopathy which leaves them very debilitated,” adding, “ and they die within five years typically on average of onset.”

Patisiran blocks the synthesis of that mutant transthyretin and it was approved in 2018 to treat the neuropathy associated with hereditary ATTR.

The company also hopes to get the drug approved to treat the cardiopathy associated with hereditary ATTR. Dr. Vaishnaw said the company is thrilled that the drug is getting to the patients to help their neuropathy but also by the “preliminary results for the cardiomyopathy which we are now firming up with additional studies in patients that have a lot of the hereditary ATTR cardiomyopathy. And that way, we look to help the full spectrum,” adding, “We’re excited about that the way doctors have embraced what in their eyes looks to be a very important therapy for this disease.”

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