US-based Alnylam Pharmaceuticals has signed an exclusive agreement with Medison Pharma to commercialise its RNAi therapeutic Onpattro in Israel.
Onpattro secured US and European regulatory approval in August last year for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy.
The drug, which is meant for intravenous administration, is designed to target and silence specific messenger RNA in order to block the production of TTR protein.
It blocks the transthyretin production in the liver, thereby decreasing accumulation in the body tissues and slowing down disease progression. The drug is yet to obtain approval for use in Israel.
Alnylam Pharmaceuticals senior vice-president Theresa Heggie said: “Our partnership with Medison marks an important step in our global commercial expansion and signals our intent to ensure that patients suffering from serious rare diseases have access to our medicines, regardless of location.
“Medison has a strong organisation with a proven track record of commercialising orphan products successfully, together with an infrastructure uniquely suited to supporting patients suffering from rare diseases in Israel and providing access to our potentially transformational therapies.”
The agreement also covers commercialisation of Alnylam’s other late-stage investigational RNAi candidates, givosiran and lumasiran.
Givosiran is being developed to treat acute hepatic porphyria (AHP), while lumasiran is intended for Primary Hyperoxaluria Type 1 (PH1).
Medison Pharma founder and CEO Meir Jakobsohn said: “Alnylam’s portfolio of Onpattro and potentially ground-breaking medicines in late stage development will strengthen our rare disease portfolio, fulfilling Medison’s vision to provide innovative treatments to patients in Israel.
“Patients with hATTR amyloidosis with polyneuropathy in Israel deserve to have the earliest possible access to novel new treatments and we look forward to making this a reality, beginning with Onpattro.”