ISTOCK, BOJAN89Alnylam Pharmaceuticals is reporting positive results from a clinical trial using patisiran—an RNA interference (RNAi) drug—to treat patients suffering from hereditary amyloidosis with polyneuropathy, a life-threatening genetic disorder. Alnylam has announced it plans to file for approval in the U.S. by the end of the year. If this goes through, patisiran would become the first RNAi therapeutic on the market.
In the Phase 3 clinical trial “Apollo,” more than 90 percent of 148 patients who were administered patisiran showed a halt in disease progression, compared to 4 percent who showed an improvement among 77 placebo patients. More than half of patisiran patients also showed significant improvements in sensory, motor, and autonomic neuropathy symptoms and reported overall improvements in quality of life. On a scale of neurological disease impairment, scores declined among treated volunteers, whereas the scores of patients administered a placebo worsened over the 18-month period. Side effects—which included swelling of extremities, diarrhea, and infusion-related reactions—were not considered significant and were between the groups.
Hereditary amyloidosis (hATTR) is a rapidly progressive and often fatal disease that affects 50,000 people globally. From symptom onset, patients have ...
