Alnylam Pharmaceuticals and Gen Sign Distribution Agreement in Turkey for ONPATTRO® (patisiran), the First-in-Class ‘Gene-Silencing’ RNAi Therapeutic
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Gen, a GMP-certified pharmaceutical company specializing in rare diseases, today announced an exclusive Distribution Agreement for ONPATTRO®, a first-in-class RNAi therapeutic for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.
“Our partnership with Gen enables us to extend access to ONPATTRO to patients suffering from hereditary ATTR (hATTR) amyloidosis with polyneuropathy in Turkey where we currently don’t have a presence,” said Brendan Martin, Vice President and Acting Head of Canada, Europe, Middle East and Africa, Alnylam Pharmaceuticals. “There are a significant number of patients in Turkey who urgently need new treatment options and we are delighted to partner with Gen to bring ONPATTRO to those in need.”
Abidin Gülmüs, CEO of Gen stated: “We are proud of our reputation as one of Turkey's leading specialty pharmaceutical companies and are excited to have partnered with Alnylam. Through collaborations with leading international companies, we aim to bring innovative medicines to patients in Turkey in the fastest and most reliable way possible.”
Patients in Turkey were among those who participated in the randomized, double-blind, placebo-controlled, global Phase 3 APOLLO study, the largest-ever study in hATTR amyloidosis patients with polyneuropathy, which led to the approval of ONPATTRO in the U.S. and EU in 2018.
About ONPATTRO® (patisiran)
ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. Based on Nobel Prize-winning science, ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO 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 polyneuropathy associated with the disease. For more information about ONPATTRO, visit ONPATTRO.com.
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. Hypotension, which may include syncope, has also been reported as a symptom of IRRs.
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.
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole 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 (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Brazil and Switzerland, and GIVLAARI® (givosiran), approved in the U.S and the EU. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in late-stage development. Alnylam is executing on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA.
Gen is the fastest growing pharmaceutical company in Turkey. Teamed up with its leading international partners and compliant with ethical and scientific principles, Gen supplies products used in treatment of rare diseases and disorders in different branches and aims to bring these products to patients in the easiest, fastest and most reliable way possible while striving to find and bring new treatments to patients with unmet medical needs. With its GMP certificated production facility based in Ankara, Gen exports its products to different countries and has offices in Ankara (HQ), İstanbul, İzmir, Trabzon, Azerbaijan, Kazakhstan and Russia with 400+ employees. For more information please visit the Gen website.
Alnylam Forward Looking Statements
Various statements in this release concerning future expectations, plans and prospects, including, without limitation, Alnylam's views and plans with respect to the ability to extend patient access to ONPATTRO in Turkey through the announced Distribution Agreement with Gen, and the number of patients in Turkey within the approved indication for ONPATTRO who are in need of new treatment options, Gen’s views and plans with respect to the speed and reliability with which it is able to bring innovative medicines to patients in Turkey, and Alnylam’s expectations regarding the continued execution on 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; 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 or its marketed products, including ONPATTRO in Turkey; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including lumasiran and product candidates developed in collaboration with others, including inclisiran, and maintaining regulatory approval and obtaining pricing, reimbursement and access for its products, including ONPATTRO and GIVLAARI; progress in continuing to establish a commercial and ex-United States infrastructure, including in Europe; successfully launching, marketing and selling its approved products globally, including ONPATTRO and GIVLAARI, and achieve net product revenues for ONPATTRO within its expected range during 2020; potential risks to Alnylam’s business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom, including without limitation, any risks affecting access to ONPATTRO in Turkey, Alnylam’s ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the ranges of its expected guidance and achieve a self-sustainable financial profile in the future, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products, and Ironwood, for assistance with the education about and promotion of GIVLAARI in the U.S.; the outcome of litigation; the risk of government investigations; and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)
+44 1628 244960
Ayhan Yener, MD
+90 554 566 57 40
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Esri Offers Students Free Access to Software for Continued Education Through Coronavirus Closures30.3.2020 16:29:00 EEST | Press release
Esri, the global leader in location intelligence, today announced that it will provide free access to its ArcGIS platform and learning resources through the Learn.ArcGIS.com website to support college and university students who no longer have access to campus computer labs during the COVID-19 outbreak. Students will receive access to ArcGIS Online and over 20 apps including ArcGIS Pro, along with a library of lessons to continue their learning and complete courses. Access is available globally to students ages 18 and over. Learn ArcGIS promotes learning by doing through guided lessons based on real-world problems in industries such as urban planning and disaster response, and new lessons on public health have just been added. Learn ArcGIS lessons cover both beginner and advanced topics and are available in seven languages. “We believe education creates opportunities for people of all ages, while building communities and advancing our knowledge for a better world,” said Jack Dangermond
Aptorum Group Collaborates with Covar Pharmaceuticals To Investigate at Least 3 Repurposed Drug Candidates (SACT-COV19) for Coronavirus Disease 2019 (COVID-19) under Existing Smart-ACT™ Platform and Acticule Infectious Disease Platform30.3.2020 15:00:00 EEST | Press release
Aptorum Group Limited (Nasdaq: APM) ("Aptorum Group"), a biopharmaceutical company focused on the development of novel therapeutics for unmet needs including but not limited to infectious, orphan and metabolic disease areas, today announced that it is initiating an additional research and development project targeting the coronavirus group and has completed initial screening under the Smart-ACT™ platform, a repurposed and new drug discovery platform, to select, out of more than 2,600 approved small drug molecules, at least 3 potential candidates for further preclinical investigation against the new coronavirus disease, COVID-19. Aptorum Group is collaborating with Toronto based Covar Pharmaceuticals and has also entered into agreement with the University of Hong Kong’s Microbiology Department to conduct further preclinical investigation of the selected candidates prior to seeking approval from regulatory agencies to initiate clinical trials on suitable candidates. In particular, Aptoru
Daiichi Sankyo Submits Application for CAR T Therapy Axicabtagene Ciloleucel for Treatment of Patients with Certain Relapsed/Refractory B-cell Lymphomas in Japan30.3.2020 15:00:00 EEST | Press release
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced that it has submitted a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for chimeric antigen receptor (CAR) T cell therapy axicabtagene ciloleucel for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma and related lymphomas. In January 2017, Daiichi Sankyo received exclusive development, manufacturing and commercialization rights for axicabtagene ciloleucel in Japan from California-based Kite, a Gilead Company. The Japan NDA submission is based on previous pivotal trial data conducted globally by Kite for axicabtagene ciloleucel in addition to results from a phase 2 bridging study conducted by Daiichi Sankyo in Japan. Both trials included patients with four aggressive types of relapsed/refractory B-cell lymphomas including diffuse large B cell lymphoma (DLBCL); primary mediastinal B-cell lymphoma (PMBCL); transformed follicular lymphoma (TF
HCL Technologies COVID-19 Status Update30.3.2020 11:40:00 EEST | Press release
HCL Technologies Ltd (HCL) has been monitoring the Covid-19 outbreak since late January and had invoked its Business Continuity Plan and Risk Management Framework quite early to minimize the impact on its employees and clients. The situation arising out of this outbreak and its rapid spread across the world is unprecedented and is extremely dynamic. The economic impact is visible with varying degrees of intensity across multiple countries where we operate. HCL has had a well-defined and structured pandemic policy in place which is resilient, pragmatic and accords paramount importance to employee safety & health and client commitments. We are actively engaged with our clients and have executed the client-specific and HCL internal operations-centric Business Continuity Plan that ensures continuity in delivering to our client and employee commitments. HCL is constantly reviewing the situation at hand with utmost priority and fully complying with all government advisories and recommendatio
GSMA Report Highlights That Mobile Money Accounts Now Exceed One Billion Worldwide30.3.2020 11:00:00 EEST | Press release
The GSMA today unveiled the annual ‘State of the Industry Report on Mobile Money’, offering a view of the mobile money landscape and highlighting the impact that greater financial inclusion has on lives, economies and innovation, especially in emerging markets. This year’s report looks at what one billion registered accounts means for the mobile money industry, mobile money users and the future of the mobile money ecosystem. It provides a comprehensive picture of adoption and usage around the globe. Significantly, the report highlights that 2019 marked a major milestone for the mobile money industry, with over one billion registered accounts and close to two billion dollars in daily transactions. For the first time, digital transactions represented the majority, 57 per cent, of mobile money interactions. The industry is witnessing increasing user trust and relevance. With 290 live services in 95 countries and 372 million active accounts, mobile money is entering the mainstream and beco
Rewired Targets Growth-Stage AI Ventures30.3.2020 10:00:00 EEST | Press release
Rewired, the robotics focused venture studio that invests in growth-stage technology ventures, is seeking to add new artificial intelligence and robotics investments to its portfolio. The portfolio currently encompasses innovations in machine learning, robotics, bionics, smart sensors, mapping and localization in the UK and USA. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200330005016/en/ Backed by investors who believe that analysts are underestimating the prospects of AI, Rewired seeks to make investments of between $5m and $30m. Its status as a venture studio means that Rewired uniquely seeks to attain a substantial or majority position as an investor, and to then provide long term funding and expertise. The Rewired approach is to alleviate founders from the burden of constant fundraising so they can focus on growth, whilst avoiding the dilution that VC’s typically accept during multiple funding rounds. Rewired is seek
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom