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 dont 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 Glms, 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 bodys 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.
Story continues
Important Safety Information (ISI) for ONPATTRO
Infusion-Related Reactions
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).
Adverse Reactions
The most common adverse reactions that occurred in patients treated with patisiran were peripheral oedema (30%) and infusion-related reactions (19%).
About RNAi
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 Alnylams RNAi therapeutic platform, function upstream of todays 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.
About Alnylam
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. Alnylams 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.
About Gen
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), stanbul, zmir, 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, Gens views and plans with respect to the speed and reliability with which it is able to bring innovative medicines to patients in Turkey, and Alnylams 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 Alnylams 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, Alnylams 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005133/en/
Contacts
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom(Investors and Media)+1-617-682-4340
Fiona McMillan(Media, Europe)+44 1628 244960
Gen Ayhan Yener, MD(Medical Director)+90 554 566 57 40
View post:
- IOM not webcast today. Why Not? [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- National Academies skeptical at Best. [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Some Confusion Exists [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Why DTC Genomics IS Medicine. [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- First Mari, Now Linda. Who's next? [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Is it true? [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Re-Reviewing the National Academies [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- The problem with nonclinicians....... [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Crazy Night of Emails to Government [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Adrienne Carlson's Personalized Medicine. [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Tell Me, How do you feel now? Sherpa's RX [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- This Just In. 23andMe to go to GPs. I love my readers!! [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Sorry so long away [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- 2D6 Rears its ugly head..... [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Ok, Fine, Back to Plavix [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Kaiser a protoype for Collins' Aim [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- A few months late to the party.... [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Stated Another Way....... [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Excuse Me? Harvard and Navigenics? WTF? [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Follow up to Yesterday's WTF? Harvard, Navi? and Pfizer??? [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Did you get your kit? Thanks Dr. Rob from MedCo [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Gluco...Wha? Parkinson's Disease and Glucocerebrosidase mutations. [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Away and now back, What did I miss???? 23andme layoffs? Selling Genomes for cheap up next! [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Change IS Needed. I agree with William, sometimes. [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Good Enough Science? Apparently so at 23andme [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Long QT Syndrome, location matters [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- Congratulations Generation Health. Nice pick up! [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- An argument 23andSerge can't win...23andme but not medicine [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- Stop. Breathe. Repeat. An analysis of the direction of DTC Genomics Field. [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- Hey DTC genomics, Stay Private, Stay Alive, Go Public and Die [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- You can't have it both way. Either scared your genome is sold off or not. [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- 15 Days Away Gives Time for Perspective. [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- What about the SACGHS registry? Another missed opportunity? [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- AJHG is in and my Favorite Muin is in it! But He Is NOT the Father! [Last Updated On: December 13th, 2009] [Originally Added On: December 13th, 2009]
- Navigenics for 23andMe prices? [Last Updated On: December 18th, 2009] [Originally Added On: December 18th, 2009]
- Lp(a) Maybe there's something there that wasn't there before? [Last Updated On: December 24th, 2009] [Originally Added On: December 24th, 2009]
- Another Year, Another Bankruptcy [Last Updated On: December 31st, 2009] [Originally Added On: December 31st, 2009]
- 5 Technologies going bye bye in this decade? [Last Updated On: January 6th, 2010] [Originally Added On: January 6th, 2010]
- Hackers, HITECH and HIPAA in DTC Genomics, Oh My! [Last Updated On: January 7th, 2010] [Originally Added On: January 7th, 2010]
- Personal Genomics Flop.....big Belly Flop! [Last Updated On: January 8th, 2010] [Originally Added On: January 8th, 2010]
- Gotta Love It. Even the daycare....... [Last Updated On: January 11th, 2010] [Originally Added On: January 11th, 2010]
- Congratulations Navigenics. You ARE a clinical lab! Uh-Oh... [Last Updated On: January 12th, 2010] [Originally Added On: January 12th, 2010]
- CETP, Jewish Centenarians and Alzheimers [Last Updated On: January 14th, 2010] [Originally Added On: January 14th, 2010]
- Enter the "Not" DTC Genomics Rep [Last Updated On: January 17th, 2010] [Originally Added On: January 17th, 2010]
- Why Dr. Vanier's Navigenics appointment is good for PM [Last Updated On: January 22nd, 2010] [Originally Added On: January 22nd, 2010]
- Holy Crap! MedCo Follows in CVS footsteps [Last Updated On: February 3rd, 2010] [Originally Added On: February 3rd, 2010]
- FDA, Warfarin, still not as sexy to me. [Last Updated On: February 5th, 2010] [Originally Added On: February 5th, 2010]
- Hype, Hype, Hype from a single study. [Last Updated On: February 11th, 2010] [Originally Added On: February 11th, 2010]
- I love my readers, even Renata M! [Last Updated On: February 17th, 2010] [Originally Added On: February 17th, 2010]
- How can insurers use DTC genomics to profile? [Last Updated On: February 17th, 2010] [Originally Added On: February 17th, 2010]
- 9p21.....ahem. Paynter et.al. Smackdown. Again. [Last Updated On: February 18th, 2010] [Originally Added On: February 18th, 2010]
- Hey! It's Pete Hulick! Are you Going to GET? [Last Updated On: February 19th, 2010] [Originally Added On: February 19th, 2010]
- I was wrong......AHEM [Last Updated On: February 28th, 2010] [Originally Added On: February 28th, 2010]
- G2C2, finally a tool for genomic education! [Last Updated On: March 2nd, 2010] [Originally Added On: March 2nd, 2010]
- Just 4 million? What 23andMe is worth. [Last Updated On: March 5th, 2010] [Originally Added On: March 5th, 2010]
- What a difference a year makes [Last Updated On: March 9th, 2010] [Originally Added On: March 9th, 2010]
- ........DTC Genomic Medicine? [Last Updated On: March 12th, 2010] [Originally Added On: March 12th, 2010]
- The FDA, 2c19 and the ACC [Last Updated On: March 13th, 2010] [Originally Added On: March 13th, 2010]
- The problem with Comparative Whole Genomics...... [Last Updated On: March 13th, 2010] [Originally Added On: March 13th, 2010]
- BRCA testing by 23andME is the same as Myriad Genetics. [Last Updated On: March 15th, 2010] [Originally Added On: March 15th, 2010]
- The Argument Against DTC Genomics Marketing and such [Last Updated On: March 16th, 2010] [Originally Added On: March 16th, 2010]
- A moment of Clarity. Some DTCG is not bad. [Last Updated On: March 18th, 2010] [Originally Added On: March 18th, 2010]
- SNPs for breast cancer risk? It Depends. [Last Updated On: March 18th, 2010] [Originally Added On: March 18th, 2010]
- How can MDVIP use Navigenics Test for Medicine? [Last Updated On: March 18th, 2010] [Originally Added On: March 18th, 2010]
- Why did P&G invest in Navigenics? [Last Updated On: March 23rd, 2010] [Originally Added On: March 23rd, 2010]
- PGx in DTCG? Doesn't stand up to Useful testing. [Last Updated On: March 25th, 2010] [Originally Added On: March 25th, 2010]
- End of Gene Patents? [Last Updated On: March 29th, 2010] [Originally Added On: March 29th, 2010]
- Sherpa Accepting Chief Medical Officership [Last Updated On: April 3rd, 2010] [Originally Added On: April 3rd, 2010]
- The Rumors of My Death........ [Last Updated On: April 20th, 2010] [Originally Added On: April 20th, 2010]
- Happy DNA Day! [Last Updated On: April 25th, 2010] [Originally Added On: April 25th, 2010]
- 99 USD, DNA day and patient letters [Last Updated On: April 25th, 2010] [Originally Added On: April 25th, 2010]
- 2C19, Navigenics and Clinical Reality. [Last Updated On: May 1st, 2010] [Originally Added On: May 1st, 2010]
- Coriell Personalized Medicine Collaborative rising [Last Updated On: May 7th, 2010] [Originally Added On: May 7th, 2010]
- Personal Genomes in Clinical Care. Quake paper is a waste! [Last Updated On: May 11th, 2010] [Originally Added On: May 11th, 2010]
- Personal Genomes in Clinical Care. Quake paper Falls Short! [Last Updated On: May 13th, 2010] [Originally Added On: May 13th, 2010]
- Last post edited by Drew [Last Updated On: May 13th, 2010] [Originally Added On: May 13th, 2010]
- GateKeeper? FCUK U! [Last Updated On: May 13th, 2010] [Originally Added On: May 13th, 2010]
- GateKeeper? F! U! [Last Updated On: May 15th, 2010] [Originally Added On: May 15th, 2010]
- Potential of genomic medicine, LOST [Last Updated On: May 19th, 2010] [Originally Added On: May 19th, 2010]
- How Bad Can a House Investigation be for DTC Genomics? [Last Updated On: May 20th, 2010] [Originally Added On: May 20th, 2010]