Alnylam Pharmaceuticals and Medison Pharma Partner to Commercialize RNAi Therapeutics in Israel
“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,” said
“We are proud to partner and collaborate with Alnylam in Israel,” said
Meir Jakobsohn, Founder and CEO, Medison Pharma. “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
The agreement between Alnylam and Medison includes ONPATTRO, approved in
the EU in
About ONPATTRO® (patisiran)
Patisiran, based on Nobel Prize-winning science, is an intravenously
administered RNAi therapeutic targeting transthyretin (TTR) for the
treatment of hereditary ATTR amyloidosis. It is designed to target and
silence specific messenger RNA, potentially blocking the production of
TTR protein before it is made. Patisiran blocks the production of
transthyretin in the liver, reducing its accumulation in the body’s
tissues in order to halt or slow down the progression of the disease. In
Important Safety Information (ISI) for ONPATTRO
Infusion-related reactions (IRRs) have been observed in patients treated with patisiran. In a controlled clinical study, 19% of patisiran-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with patisiran were flushing, back pain, nausea, abdominal pain, dyspnoea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to patisiran infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
Patisiran treatment leads to a decrease in serum vitamin A levels. Patients receiving patisiran should take oral supplementation of approximately 2500 IU vitamin A per day to reduce the potential risk of ocular toxicity due to vitamin A deficiency. Doses higher than 2500 IU vitamin A per day should not be given to try to achieve normal serum vitamin A levels during treatment with patisiran, as serum levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. including reduced night vision or night blindness, persistent dry eyes, eye inflammation, corneal inflammation or ulceration, corneal thickening or corneal perforation).
The most common adverse reactions that occurred in patients treated with patisiran were peripheral oedema (30%) and infusion-related reactions (19%).
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
Medison, Israel’s leading innovative pharmaceutical partner, is an
exclusive Israeli partner for global healthcare companies such as
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, including without limitation ONPATTRO, givosiran and lumasiran, plans to make ONPATTRO available in additional countries and to continue the filings for regulatory approval and launch of ONPATTRO for hATTR patients with polyneuropathy around the world, and expectations regarding its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing, delays, interruptions or failures in the manufacture and supply of its product candidates, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-
With the exception of ONPATTRO (patisiran), none of Alnylam’s
investigational therapeutics have been approved by the
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)
+44 1628 244960
Vice President, Corporate Development
+972 (0) 39250374