US-based Alnylam Pharmaceuticals, the world's leading RNAi therapeutics company, has updated on the global commercialisation of ONPATTRO (patisiran) but is still to disclose any plans for an Australian launch.
ONPATTRO is an intravenously administered RNAi gene therapy for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.
It is thought hATTR amyloidosis affects around 50,000 people worldwide.
ONPATTRO works by delivering the RNAi and reducing the production of defective transthyretin in patients with hATTR amyloidosis. This reduces the formation of amyloids and relieves symptoms.
RNAi therapeutics, which were first discovered in 1998, are an entirely new class of therapies. By harnessing RNAi, researchers can design therapies that target mutated genes and turn off the production of faulty proteins that cause disease.
Alnylam reported global revenues of US$46.1 million for the three months to the end of September, including the first sales in Japan and a regulatory filing in Brazil.
“In the third quarter of 2019 and recent period we saw strong execution on the global commercialization of ONPATTRO. For the rest of the year and beyond, we expect steady and continued growth in patients on ONPATTRO therapy through improved disease awareness, new patient finding, expansion in global markets – such as our recent launch in Japan and NDA filing in Brazil – and the potential for future label expansion in hereditary and wild-type ATTR cardiomyopathy through our recently initiated APOLLO-B Phase 3 study," said CEO Dr John Maraganore.
"We’re also pleased to have received a Priority Review and Accelerated Assessment for givosiran from the FDA and EMA, respectively, and we are preparing for the potential launch of our second RNAi therapeutic in the coming months, assuming positive regulatory reviews.
“While executing on our commercial objectives, we also made excellent progress on our late stage pipeline, including positive Phase 3 results for inclisiran announced by our partner, The Medicines Company, representing what we believe is a landmark event for Alnylam and for the entire field of RNAi therapeutics.
"In the final months of 2019, we aim to extend this encouraging track record with pivotal data from our lumasiran program, continued enrollment in our HELIOS-A Phase 3 trial and initiation of the HELIOS-B Phase 3 study with vutrisiran. Each of these anticipated milestones will bring us closer to achieving our Alnylam 2020 goals of building a multi-product, global biopharma company with a deep clinical pipeline to fuel future growth and a robust product engine for sustainable and organic innovation, a profile rarely achieved in our industry."
Alnylam Pharmaceuticals has 26 offices in 17 countries. It has no commercial presence in Australia but does have some clinical trial sites. The company's only office in the southern hemisphere is in Brazil.
The situation for ONPATTRO in Australia is somewhat complicated by the fact Sanofi had local rights to ONPATTRO until January 2018. Alnylam regained full global rights to the therapy in January 2018. In return, it granted Sanofi right to its haemophilia candidate fitusiran.