, Alnylam reported that the Phase 3 APOLLO clinical trial (NCT01960348) evaluating its drug patisiran, intended to treat hereditary ATTR (hATTR) amyloidosis with polyneuropathy, had met its primary endpoint.
Based on the results, Alynylam has initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). The rolling submission allows completed portions of an NDA to be reviewed by the FDA on an ongoing basis.
ATTR amyloidosis is a rapidly progressive genetic disease that results in the misfolding of transthyretin (TTR) proteins. Eventually, the misfolding encourages the development of amyloid fibrils that can potentially be deposited into different organs. Patisiran, an investigational RNAi therapeutic, targets TTR, thus reducing accumulation of the misfolded protein.
The disease is characterized by a clinical presentation that includes sensory and motor, autonomic and cardiac symptoms.
As of this morning, Alnylam had submitted the non-clinical and chemistry, manufacturing and controls components of the application, and expects to submit the final clinical data component by the end of 2017. Additionally, the company has requested a priority review of the application, which, if granted, will reduce the review process to 6 months.
“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,” said Eric Green, Vice President and General Manager of the company’s TTR program in a press release
. “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.”
The hope of patisiran by those developing is that it can enable the clearance of TTR amyloid deposits in peripheral tissues, restoring function to them and essentially reversing the effects of prior damage.
Alnylam has been working on patisiran in conjunction with Sanofi Genzyme, and the pair plans to submit a Marketing Authorisation Application to the European Medicines Agency by the end of 2017. Sanofi Genzyme is preparing to submit regulatory filings for patisiran in Japan and Brazil, among other countries, in the first quarter of 2017.
As currently constituted, the terms of the agreement between the two companies state that Alynylam will commercialize the therapy in the U.S., Canada and Western Europe, while Sanofi Genzyme commercializes it throughout the rest of the world.
“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,” said Muriel Finkel, president of the Amyloidosis Support Group.
On Monday, the European Medicines Agency granted Alnylam Accelerated Assessment
for patisiran, and Johns Hopkins professor of neurology Michael Polydekis, M.D.
is on the record as calling the therapy “life-changing.”
For more on patisiran and other potential breakthrough therapies from within the rare disease community, follow Rare Disease Report