The US Food and Drug Administration has granted Breakthrough Therapy designation (BTD) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) with polyneuropathy.
The drug is under development by US biotech firm Alnylam Pharmaceuticals (Nasdaq: ALNY), which has been riding high since presenting strong new data from its Phase III APOLLO study into patisiran at the European ATTR Amyloidosis Meeting in Paris. New data on Ionis Pharmaceuticals’ (Nasdaq: IONS)rival hATTR therapy inotersen were also presented at the Paris meeting, but showed a little lower than 20 point improvement after 15 months, according to the same metric as used by Alnylam.
Alnylam’s shares were barely moved by the latest news, but that’s not too surprising, given they are up more than 230% so far this year.
BTD is intended to expedite the development and review of drugs for the treatment of serious or life-threatening conditions based on clinical evidence indicating that the drug demonstrates substantial improvement on clinically significant endpoints over available therapy.
Earlier this month, the European Medicines Agency granted an accelerated assessment for patisiran for the treatment hATTR amyloidosis.
Rolling NDA initiated
Alnylam has initiated a rolling New Drug Application (NDA) for patisiran and expects the last submission by the end of 2017. Patisiran previously received Fast Track designation in the USA. Alnylam, in alliance with French pharma major Sanofi (Euronext: SAN), intends to file a marketing authorization application in the European Union around year-end.
“Hereditary ATTR amyloidosis is an aggressive, rapidly progressing, debilitating and fatal disease, and the need for new treatment options is urgent. The robust evidence provided by the APOLLO Phase III study demonstrated the potential of investigational patisiran to transform the lives of people with hATTR amyloidosis,” said Eric Green, vice president and general manager of the TTR program. “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,” he added.
Pending regulatory approvals, Alnylam will commercialize patisiran in the USA, Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world. Sanofi expects to make regulatory filings in Japan, Brazil and other countries in the first half of 2018.