Alnylam Pharmaceuticals has started its submission of a rolling new drug application (NDA), asking the U.S. Food and Drug Administration (FDA) to approve patisiran for the treatment of hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP).
The rolling submission allows the company to submit pieces of the required documentation on a rolling basis, potentially speeding the review process.
Meanwhile, with its partner Sanofi Genzyme, Alnylam plans to submit an application for approval to European authorities by year’s end.
“The initiation of the NDA filing marks an exciting moment in Alnylam’s 15-year journey — the company’s first NDA, and the first-ever application for regulatory approval of an RNAi therapeutic,” Eric Green, vice president and general manager of the TTR program at Alnylam, said in a press release.
Patisiran is a therapy that silences the TTR (transthyretin) gene by blocking the production of messenger RNA — intermediate factors in the process of making a protein from a gene. This class of drugs is called RNAi therapeutics.
In September 2017, Alnylam and Sanofi Genzyme announced that a Phase 3 trial, called APOLLO (NCT01960348), demonstrated that patisiran reduced impairment caused by the disease and improved patients’ quality of life.
The study included patients with 39 different disease-causing mutations, demonstrating that the treatment is broadly applicable.
“With the recent APOLLO Phase 3 study results, we believe that patisiran is poised to potentially become an important option for the treatment of hATTR amyloidosis, a rapidly progressive, debilitating and often fatal disease,” said Green.
“Commencing the NDA submission for patisiran is wonderful news for the hereditary amyloidosis community. We are deeply encouraged by the potential impact patisiran can make on the lives of people living with hATTR amyloidosis,” added Muriel Finkel, president of the Amyloidosis Support Groups.
“With patisiran moving one step closer to potentially being available to patients in the U.S., we can now have hope for a promising treatment option for this disease.”
Alnylam is in charge of bringing patisiran to the market in the U.S., Canada, and Western Europe, while Sanofi Genzyme works in the rest of the world. Sanofi is preparing to file applications for patisiran in Japan, Brazil and other countries in 2018.