Sanofi and Alnylam have reported positive Phase III results for their RNAi drug patisiran and plan to launch it in the US and Europe in 2018.

Results from the APOLLO Phase 3 study are in. The study recruited 225 patients suffering from hereditary ATTR amyloidosis with polyneuropathy to test patisiran, showing that the RNAi drug could improve neuropathy impairment and quality of life compared to placebo after 18 months. Detailed results will be presented at the European ATTR Amyloidosis Meeting in Paris this November.

These results makes patisiran the first RNAi drug to deliver positive Phase III results, and will support Alnylam and Sanofi’s applications for marketing approval in the US and Europe, which are planned for late 2017 and early 2018, respectively. The team expects to launch patisiran in both regions during 2018.

This would not only make patisiran the first RNAi drug in the market, but a first effective treatment for hATTR amyloidosis, a rare disease that affects around 50,000 people worldwide. There are currently no treatments approved in the US, and in Europe they’re limited to tafamidis, only in early stages of the disease and not always effective.

hATTR amyloidosis is caused by mutations in the transthyretin (TTR) gene that result in amyloid deposits in multiple tissues, especially affecting peripheral nerves and the heart. Patisiran consists of a RNAi sequence against transthyretin that induces the degradation of the mRNA encoding it before the cell can produce the mutated form of the protein. (Read more about it in my review of RNA-based therapies.)

Sanofi partnered with Alnylam back in 2014 with a $700M (€590M) inversion. For both partnered products, patisiran and fitusiran — whose trial is currently on hold after a patient death — Alnylam keeps commercialization rights in the US, Canada and Western Europe, while Sanofi gets the rest of the world. The big pharma is already preparing to file for approval with patisiran in several countries, including Japan and Brazil, in the first half of 2018.

Sanofi and Alnylam might soon have competition for patisiran; Ionis Pharmaceuticals and its partner GSK completed in May a Phase III trial in amyloid polyneuropathy with inotersen, an antisense drug. Still, analysts are expecting patisiran to take an 80% share of the market.

To further support its therapy, Alnylam is setting up a free-of-charge genetic testing and counseling program in the US, Alnylam Act, for people that suspect to have hATTR or have a family history. The biotech also announced to be considering a similar plan in Europe during an investor call yesterday.


Saibarakova Ilona, Sentavio /Shutterstock

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