Alnylam Pharmaceuticals Inc. said its drug to treat patients suffering from a rare genetic disease met the main goal in a late-stage study, moving the company closer to filing a marketing application for the drug in late 2017.
The company’s shares soared 22.2 percent to $91.70 in premarket trading on Wednesday.
The drug, patisiran, was being evaluated for the treatment of hereditary ATTR (hATTR) amyloidosis patients with polyneuropathy, which affects about 50,000 people worldwide, Alnylam said.
Polyneuropathy, a symptom of the disease, is the simultaneous malfunction of many peripheral nerves in the body resulting in tingling, numbness and kidney dysfunction.
The only approved treatments for hATTR are liver transplantation for early-stage disease and tafamidis, a drug approved in Europe, Japan and certain countries in Latin America, the drugmaker said.
Under an agreement, Alnylam said it would advance patisiran in the United States, Canada and Western Europe while French drugmaker Sanofi would commercialize it in the rest of the world.
The study, which tested patisiran against a placebo, and evaluated nerve impairment at 18 months, showed significant overall disease improvement.
Earlier this month, shares slumped after Alnylam stopped giving doses of its fitusiran drug for a rare bleeding disorder to patients enrolled in clinical studies after one patient died.