Breaking News
February 24, 2018 - UCLA researchers use fluorescent colored proteins to trace origin of heart cells
February 24, 2018 - Home Routines Can Boost a Child’s Readiness for School
February 24, 2018 - FDA Investigating Misuse, Abuse of Gabapentinoids
February 24, 2018 - Scientists find key proteins control risk of osteoarthritis during aging
February 24, 2018 - Izon announce the launch of the qEV2 and qEV10 Exosome Isolation columns
February 24, 2018 - New CSIRO technology can create clean drinking water
February 24, 2018 - Treating sleep-disordered breathing may improve prognosis of heart failure patients
February 24, 2018 - More boys begin school a year late than girls, study finds
February 24, 2018 - Early life exposure to green space could have beneficial effects on cognitive function
February 24, 2018 - Joint Surgery: Aspirin Equals NOAC for Post-Acute VTE Prevention
February 23, 2018 - Scientists identify new marker of arthritis in mice
February 23, 2018 - Beetroot juice supplements may benefit patients with heart failure
February 23, 2018 - New study identifies novel molecular biomarkers of preeclampsia
February 23, 2018 - Researchers discover new link between gut bacteria and obesity
February 23, 2018 - Aimmune Therapeutics’ Pivotal Phase 3 PALISADE Trial of AR101 Meets Primary Endpoint in Patients With Peanut Allergy
February 23, 2018 - Improving Glaucoma Care: Ophthalmology Times
February 23, 2018 - Preventing dementia: The promising, the disappointing and the inconclusive
February 23, 2018 - Duke researchers show how to retrain immune system of peanut-allergy mice
February 23, 2018 - Older males could live longer with light intensity exercise, study suggests
February 23, 2018 - C-sections and gut bacteria linked to childhood obesity risk
February 23, 2018 - Asthmatics have lower degree of DNA methylation in certain immune cells
February 23, 2018 - Uniforms coated with copper nanoparticles could reduce spread of hospital infections
February 23, 2018 - What Not to Do If You Have Asthma and Want to Get Pregnant
February 23, 2018 - Deep brain stimulation may be promising Alzheimer’s treatment
February 23, 2018 - AMSBIO offers new PARPtrap Assay Kit
February 23, 2018 - New study identifies mechanisms to lengthen egg viability in worms
February 23, 2018 - Interventions to improve self-concept could be critical in treating mental health patients
February 23, 2018 - Few minutes of physical activity may lower risk of death in older men, research suggests
February 23, 2018 - Modifications in HIV test enable rapid detection of Zika virus, study states
February 23, 2018 - Could Hackers Target Heart Devices?
February 23, 2018 - Kids’ Mental Health Status Not a Factor in Gun Storage
February 23, 2018 - Revellers ready for festival drug checks, study finds
February 23, 2018 - Stanford researchers explore how enzyme changes and becomes antibiotic-resistant
February 23, 2018 - Scientists decode molecular structure of healthy huntingtin protein
February 23, 2018 - Efficacy of cancer immunotherapy increased by deactivating tumor defence mechanism
February 23, 2018 - Epigenetic changes due to normal aging process linked to cancer risk
February 23, 2018 - Evaluations of Medicaid experiments by states, CMS are weak, GAO says
February 23, 2018 - Biomarkers ID’d for Anesthesia-Related Neural Damage
February 23, 2018 - Study reveals how kidney disease happens
February 23, 2018 - Iron deficiency early in life can have long-lasting consequences for the brain
February 23, 2018 - High protein diet reduces risk of Alzheimer’s disease, research finds
February 23, 2018 - Research extends powerful capabilities of MRI to nanometer scale
February 23, 2018 - Scientists show that cutting-edge technique can efficiently sort nano-sized particles
February 23, 2018 - Dornier’s new laser innovation delivers confidence to glide through deflected scopes
February 23, 2018 - Neurocrine Biosciences Will File New Drug Application for Opicapone for Parkinson’s Disease Based on Existing Pivotal Clinical Trial Data
February 23, 2018 - Bariatric Surgery Enabled Stopping Diabetes Meds
February 23, 2018 - C-sections and gut bacteria increase risk of childhood obesity
February 23, 2018 - Busting myths about diet and kidney stones
February 23, 2018 - Reformulating vaccines to prevent relapse
February 23, 2018 - Prophylactic use of haloperidol does not reduce delirium burden
February 23, 2018 - Higher prevalence of kidney stones in Southern United States
February 23, 2018 - Lithotripsy has revolutionized modern kidney stone management
February 23, 2018 - Researchers describe important step toward gene therapy for patients with Sandhoff disease
February 23, 2018 - Pain Therapeutics Resubmits New Drug Application for Remoxy ER, an Abuse-Deterrent, Extended-Release Drug Candidate for the Treatment of Chronic Pain
February 23, 2018 - Combo Therapy Highly Active in Untreated RCC
February 23, 2018 - Boosting a key protein to help bones that won’t heal
February 23, 2018 - Advanced method holds promise for substantial improvements in breast cancer diagnosis
February 23, 2018 - MRI and machine learning could predict whether OCD patients will benefit from treatment
February 23, 2018 - Comparing low-fat and low-carb diets finds little difference
February 23, 2018 - FDA Alert: Clarithromycin (Biaxin): Drug Safety Communication
February 23, 2018 - Out of Limbo Into Bomb Scare
February 23, 2018 - Patients who achieve short-term weight loss before bariatric surgery have better outcomes
February 23, 2018 - Beetroot may reduce kidney failure risk after heart x-ray, research reveals
February 23, 2018 - Sleep disruptions in menopause correlated with hot flashes and depression
February 23, 2018 - Scientists discover new treatment approach to curb severe myocarditis
February 23, 2018 - ‘Click chemistry’ approach may improve disease-fighting properties of drugs
February 23, 2018 - NIGHTSEA and EMS team up to offer KEY Award in fluorescence stereo microscopy
February 23, 2018 - Calorie restriction improves intestinal-tissue regeneration after injury
February 23, 2018 - Tobacco Kills, No Matter How It’s Smoked: Study
February 23, 2018 - Q&A: Avindra Nath, MD | Medpage Today
February 23, 2018 - Adherence to sleep apnea treatment affects risk of hospital readmission
February 23, 2018 - Zika virus could be alternative for treatment of aggressive brain cancer
February 23, 2018 - Carbon monoxide enhances efficacy of antibiotic against stomach infection
February 23, 2018 - Study sheds light on biological mechanisms that drive rare pediatric neurogenetic disorders
February 23, 2018 - MSD and Ferring Pharmaceuticals Complete Largest Ever Clinical Trial in Postpartum Haemorrhage
February 23, 2018 - Portable ultrasound can help better detect fluid in the lungs of patients with end-stage kidney disease
February 23, 2018 - Postnova AF2000 system offers reliable characterization of trace metal colloid distribution in the environment
February 23, 2018 - Pioneering study may pave way for effective painkillers to treat neuropathic pain
February 23, 2018 - Research opens up new avenue to minimize risks of transplants
February 22, 2018 - Cabozantinib Active in Advanced Thyroid Cancer
U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy

U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy

image_pdfDownload PDFimage_print

patisiran

Treatment for Amyloidogenic Transthyretin Amyloidosis

U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy

CAMBRIDGE, Mass.–(BUSINESS WIRE) November 20, 2017 — Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) with polyneuropathy. BTD is intended to expedite the development and review of drugs for the treatment of serious or life-threatening conditions based on clinical evidence indicating that the drug demonstrates substantial improvement on clinically significant endpoints over available therapy.

Alnylam has initiated a rolling New Drug Application (NDA) for patisiran and expects the last submission by the end of 2017. Patisiran previously received Fast Track Designation in the U.S., and was recently granted accelerated assessment by the European Medicines Agency. Alnylam, in alliance with Sanofi Genzyme, intends to file a marketing authorization application in the European Union around year-end.

“Hereditary ATTR amyloidosis is an aggressive, rapidly progressing, debilitating and fatal disease, and the need for new treatment options is urgent. The robust evidence provided by the APOLLO Phase 3 study demonstrated the potential of investigational patisiran to transform the lives of people with hATTR amyloidosis,” said Eric Green, Vice President and General Manager of the TTR program. “Breakthrough Therapy Designation enables us to work closely with the FDA on the review of the NDA to potentially bring patisiran to patients living with this devastating disease as quickly as possible.”

Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.

About Patisiran

Patisiran is an investigational intravenously administered RNAi therapeutic targeting transthyretin (TTR) in development 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. This may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues. The safety and efficacy of patisiran have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About APOLLO Phase 3 Study

The APOLLO Phase 3 study (N=225) was a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy.

About LNP Technology

Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

Alnylam – Sanofi Genzyme Alliance

In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, formed an alliance to accelerate the advancement of RNAi therapeutics as a potential new class of innovative medicines for patients around the world with rare genetic diseases. The alliance enables Sanofi Genzyme to expand its rare disease pipeline with Alnylam’s novel RNAi technology and provides access to Alnylam’s R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven global capabilities to advance late-stage development and, upon commercialization, accelerate market access for these promising genetic medicine products. In the case of patisiran, Alnylam will advance the product in the United States, Canada and Western Europe, while Sanofi Genzyme will advance the product in the rest of the world.

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 major new class of medicines, known as RNAi therapeutics, is on the horizon. 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.

About Alnylam Pharmaceuticals

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, and hepatic infectious 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 discovery platform and deep pipeline of investigational medicines, including four product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute 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 employs over 600 people in the U.S. and Europe and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

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 complete results from its APOLLO Phase 3 clinical trial for patisiran and the potential implications of such results for patients, its plans for and the expected timing of regulatory filings seeking approval for patisiran from regulatory authorities in the United States, Europe and ROW countries, its expectations regarding the potential for patisiran to improve the lives of hATTR amyloidosis patients with polyneuropathy and their families, its plans for the commercialization of patisiran if approved by regulatory authorities, 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-United States infrastructure, 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, 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 for development, manufacture and distribution of products, 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 Quarterly Report on Form 10-Q 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.

Patisiran has not been approved by the U.S. Food and Drug Administration, European Medicines Agency, or any other regulatory authority and no conclusions can or should be drawn regarding the safety or effectiveness of this investigational therapeutic.

Source: Alnylam Pharmaceuticals, Inc.

Posted: November 2017

Related Articles

patisiran FDA Approval History

Tagged with:

About author

Related Articles