Alnylam Highlights Progress on Global Market Access for ONPATTRO (patisiran)
− In Europe, Company Achieves Favorable Health Technology Assessment Ratings for ONPATTRO in Multiple Countries –
“We’re encouraged by the positive reception for global access to
ONPATTRO from many private and public payers. With 10 VBAs completed
with commercial payers in the U.S., we’re fulfilling our commitment that
ONPATTRO is reimbursed based on its ability to deliver outcomes in the
real-world setting comparable to those demonstrated in clinical trials,”
Alnylam has made significant progress securing access to ONPATTRO for multiple countries in the EU. The Company remains on track with pricing and reimbursement procedures in nearly all EU markets, with encouraging assessment outcomes and ongoing discussions with authorities. Key updates include:
Germany: Launched ONPATTRO one month following EC authorization ( October 2018). Since then, ONPATTRO has become the only product for the treatment of hATTR amyloidosis to receive a “considerable benefit” rating from the Joint Federal Committee (G-BA). France: Recognized ONPATTRO as being the only product for the treatment of hATTR with a significant public health impact as noted by its ASMR III and Intêret Santé Publique (ISP) ratings. ONPATTRO is currently reimbursed through Temporary Authorization for Use. Austria: Achieved access as of October 2018. Luxembourg: Achieved access as of October 2018. Netherlands: Reached a nationwide agreement to reimburse ONPATTRO for all eligible patients through joint negotiations coordinated by Zorgverzekeraars Nederland (ZN), the Dutch Association of Health Insurers. Sweden: Published HTA assessment from the Dental and Pharmaceutical Benefits Agencyin Sweden(TLV) allowing a subsequent ongoing negotiation with the New Therapy Council( NT- Council) to allow usage of ONPATTRO within the County Councils.
Pricing and reimbursement procedures are progressing in several
additional countries, including the
Alnylam remains committed to making ONPATTRO available and reimbursed worldwide in alignment with its Patient Access Philosophy, which outlines the Company’s commitment to access while delivering value to patients, physicians, and payers.
Physicians and patients in
Visit ONPATTRO.com for more information, including full prescribing information.
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 TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR 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
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, 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 ONPATTRO 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
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, 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. night blindness).
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.
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.
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of diseases with high unmet need. ONPATTRO® (patisiran) is the first-ever RNAi therapeutic approved by the U.S.
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 availability and reimbursement for ONPATTRO in multiple geographies 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-
Alnylam Pharmaceuticals, Inc
Christine Regan Lindenboom
(Investors and Media)