The company is weeks away from completing its rolling NDA for Patisiran.
With the finish line in sight, RNAi therapy company Alnylam got a boost of energy from the U.S. Food and Drug Administration this weekend for its hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) treatment Patisiran.
The FDA awarded Alnylam’s RNAi therapy Breakthrough Therapy Designation, which could possibly speed up the time it takes for the treatment to get to market – presuming it receives the final authorization. The company is weeks away from completing its rolling New Drug Application for Patisiran. Alnylam said in its announcement this morning that it anticipates finishing the NDA by the end of 2017. Patisiran previously received Fast Track Designation in the U.S., and was recently granted accelerated assessment by the European Medicines Agency.
The FDA gave Alnylam the new designation following the September announcement of Patisiran Phase III data for hATTR amyloidosis. Earlier this month, Alnylam and its development partner Sanofi presented that data at the 1st European ATTR Amyloidosis Meeting in Paris. During its presentation, the companies demonstrated that Patisiran showed a 34-point mean difference in the modified neuropathy impairment score at 18 months. The mean change from baseline in mNIS+7 was significantly lower in the Patisiran group as compared with placebo, Alnylam said. The Phase III trial data showed mean and median changes in mNIS+7 impairment scores for Patisiran patients were both negative values. That indicates an overall improvement in the majority of patients compared with baseline, Alnylam said. That score came with a 6-point improvement for patients, meaning that over a period of time, the patients got better. A quality of life score also demonstrated improvement for the patients in the study.
Eric Green, general manager of Alnylam’s TTR program, said the need for a treatment for Hereditary ATTR amyloidosis is urgent due to the aggressive nature of the fatal disease. Approval of Patisiran will “transform” the lives of patients with the disease, he said.
“Breakthrough Therapy Designation enables us to work closely with the FDA on the review of the NDA to potentially bring Patisiran to patients living with this devastating disease as quickly as possible,” Green said in a statement.
Hereditary ATTR amyloidosis is an inherited disease caused by mutations in the TTR gene. Those mutations cause abnormal amyloid proteins to collect and damage organs and tissues, such as cardiac muscle and peripheral nerves. The damage can lead to various problems such as peripheral sensory neuropathy, autonomic neuropathy and cardiomyopathy. Alnylam has been developing its hATTR treatment for 15 years. The drug is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made.
If approved, Alnylam will commercialize Patisiran in the U.S., Canada and Western Europe. Sanofi Genzyme will commercialize the product in the rest of the world. Sanofi will apply for marketing authorization in Europe by the end of the year, Alnylam said.
Shares of Alnylam closed at $127.51 on Friday, but did rise a bit in late trading to $128.50. Shares of Sanofi closed at $44.54 on Friday.
Alnylam is hoping to get to market before competitor Ionis Pharmaceuticals and its treatment inotersen.
