NICE backs Akcea’s Tegsedi, pipping Alnylam to hATTR okay

Pharmaphorum | April 18, 2019

NICE has recommended that Akcea’s hereditary transthyretin-related amyloidosis (hATTR) treatment Tegsedi be made available on the NHS in England, ahead of a rival therapy from Alnylam. Both Tegsedi (inotersen) and Alnylam’s rival hATTR therapy Onpattro (patisiran) were turned down by the UK healthy technology assessment (HTA) agency in draft guidance published in December on the grounds that their cost was too high to balance their clinical benefit. At full price both drugs cost around £300,000 per year per patient, but there are only around 150 patients with hATTR in the UK, according to NICE. Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association said that NICE’s decision marks “a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date.” “There is a critical need for innovative new therapies for people across the UK living with this debilitating disease. We hope inotersen will be available to patients in the UK very soon,” he added. After a confidential price cut, Akcea has persuaded NICE to change its position on Tegsedi, paving the way for the first-ever treatment for hATTR to be made available patients with the ultra-rare, progressive condition, which causes the liver to produce abnormal transthyretin protein.

Spotlight

Learn more about green chemistry and how Pfizer is reducing the use of chemicals in our research and development processes.

Spotlight

Learn more about green chemistry and how Pfizer is reducing the use of chemicals in our research and development processes.

Related News

PHARMA TECH

Vivera Receives 3rd U.S. Patent Allowance for ZICOH Drug Delivery Device

Vivera Pharmaceuticals, Inc. | May 26, 2022

Vivera is pleased to announce the allowance of its third U.S. patent for ZICOH™, the Company's high-tech, electronic dose-controlled portfolio of drug delivery devices. The patent allowance from the U.S. Patent and Trademark Office secures ZICOH's rights as a smart dosing system with automated delivery, measurement, and management for oral format medications, including pills, capsules, and tablets. ZICOH is an electronic dose-controlled delivery device and technology designed to enable real-time communication through the drug supply chain, from drug manufacturers, distributors, and wholesalers to pharmacists, providers, physicians, caregivers, and patients. The device can be programmed to dispense the medication dosage type, amount, and frequency according to the health care provider's orders to ensure compliance with dispensed prescriptions while also implementing controls to help prevent medication misuse and abuse. Later versions of the device will also implement user authentication features, such as fingerprint authorization and facial recognition, to help prevent prescription drugs from being diverted. Medication reminders are another core feature of ZICOH. These reminders can be programmed into the device by the pharmacist per the physician's orders to alert the patient when it's time to take their medication. This feature can be particularly beneficial for patients who forget to take medications on time or for patients and caregivers who manage multiple medications. "ZICOH is a major innovation in the medical device and technology sectors. This device is going to make it easier than ever for patients to comply with their medication regimens, regardless of how complex they are." Mehdi Hatamian, Ph.D., Chief Scientific Advisor for Vivera This third patent differs from the device's two previously issued patents in that it specifically pertains to the dispensing of tablets, capsules, and pills. The device has already secured its patent rights as a secure, smart liquid and inhaler delivery device with automated dose delivery, measurement, and management. "We are proud of the progress we have made on the development of ZICOH's multiplatform uses and technology," said Paul Edalat, CEO and Chairman of Vivera. "The way that Tesla revolutionized the automotive industry, and the way Apple created a category for smart devices, is what Vivera is doing to the prescription dispensing industry. ZICOH will forever change the way that medications are prescribed and dispensed, and Vivera is reaching the milestones to get there in a fraction of the time projected." Vivera is looking forward to bringing ZICOH to market. The Company has already initiated the device's design and is looking forward to having a working prototype by late summer 2022. About Vivera Pharmaceuticals Vivera Pharmaceuticals is an innovative, science-driven pharmaceutical company located in Southern California. The Company has global exclusivity to license the patented and patent-pending TABMELT® sublingual drug delivery system for pharmaceutical use and holds its own issued patents on ZICOH™, a smart dose-controlled medical device. It also has patents pending on its telemedicine station, MDZone. With multiple divisions, including its pharmaceutical, neurosciences, medical technology, biosciences, and advanced diagnostics divisions, Vivera Pharmaceuticals is vertically integrated with patented technology, manufacturing capabilities, and distribution for its products.

Read More

BUSINESS INSIGHTS

C-Path and Replica Analytics Collaboration Aims to Accelerate Rare Disease Research

Replica Analytics | June 30, 2022

Critical Path Institute and Replica Analytics, an Aetion company, announced a new partnership that involves leveraging synthetic data to further catalyze the generation of actionable solutions to accelerate drug development for rare diseases. Replica Analytics will help generate synthetic datasets across rare and orphan indications in which patient-level datasets are often quite small, which heightens considerations regarding data privacy and accessibility. Together with real data, these synthetic data will help maximize the utility of C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform which provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted to accelerate drug development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data. Once added to RDCA-DAP, the synthetic data will also represent valuable real-world, electronic health record (EHR) based data available on the platform, opening doors to modeling and research based on longitudinal focused analyses. "Our collaboration with Replica Analytics is important because synthetic data can add value to real-world data while observing data privacy considerations, which will help accelerate overall rare disease drug development. The goal of RDCA-DAP is to provide a centralized and standardized infrastructure to support and accelerate rare disease characterization and therapy development, this collaboration is in line with our efforts." RDCA-DAP Scientific Director Alexandre Betourne, Pharm.D., Ph.D Synthetic data generation is a privacy enhancing technology that has been gaining rapid adoption, particularly in the life sciences sector. SDG uses AI to create machine learning models that learn the statistical patterns and properties of real datasets to generate data that retain the same characteristics as the original dataset, but with no one-to-one mapping back to an identifiable person. SDG can help amplify small datasets, simulate virtual patients to augment patients in existing datasets, and optimize the design of small sample clinical trials. "We are certainly seeing a growing opportunity to partner with organizations like C-Path for generating datasets that are fit-for-purpose," says Khaled El Emam, Ph.D., SVP and GM of Replica Analytics. "Synthetic data, which preserves the integrity and utility of source data, as well as being privacy-protective, can be a very valuable tool to enable this collaboration." In May 2022, C-Path hosted a webinar in which the Replica Analytics team introduced SDG and discussed the role the technology can play in researching and treating rare diseases. The event was recorded and can be viewed on C-Path's YouTube channel. About C-Path Critical Path Institute is an independent, non-profit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. Critical Path Institute is supported by the Food and Drug Administration of the U.S. Department of Health and Human Services and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government sources, totaling $11,196,634. The contents are those by the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. About Replica Analytics, an Aetion company Replica Analytics is the premier science-based SDG technology provider to the healthcare industry. The company a pioneer in the development of unique technologies for generating privacy-protective synthetic data that maintain the statistical properties of real-world data. The company was acquired in late 2021 by Aetion, the leading regulatory-grade real-world evidence technology provider. Replica Synthesis software provides a full suite of synthetic data generation and evaluation capabilities that can solve multiple grand challenges facing the life sciences industry, and health research in general. About Aetion Aetion is a healthcare analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs healthcare's most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation.

Read More

BUSINESS INSIGHTS

QHP Capital Acquires Lexitas Pharma Services

Lexitas and QHP Capital | July 08, 2022

QHP Capital the management company for NovaQuest Private Equity announced its acquisition of Lexitas Pharma Services a full-service ophthalmology contract research organization supporting biopharmaceutical and medical device clinical trials. Since 2011, Lexitas has provided clinical trial and medical strategy solutions to biopharmaceutical and medical device companies developing ophthalmic products. The Company provides end-to-end support and development expertise in the anterior and posterior segments of ophthalmology. Lexitas has conducted clinical trials for dry eye disease, glaucoma, cataract, uveitis, pediatric myopia, diabetic macular edema, age-related macular degeneration, and retinitis pigmentosa, among others. The Company’s therapeutic area expertise, relationships with ophthalmic KOLs, and network of high-performing research sites has led to strong outcomes for its international customer base. “For the past decade, Lexitas has had the incredible opportunity and privilege to partner with talented companies with amazing science as they develop their ophthalmic drugs and devices. Partnering with the QHP team brings deep expertise and domain knowledge that is going to allow Lexitas to improve and increase the scope, quality, and efficiency of our capabilities. We couldn’t be more thrilled to be working with QHP and are excited at the prospect of getting safe and efficacious ophthalmic drugs to patients efficiently and quickly,” George Magrath, M.D., CEO of Lexitas “Lexitas is an excellent fit with QHP’s philosophy and strategy of providing strategic capital to growing clinical trial services providers in niche, specialized therapeutic areas,” said Vern Davenport and Jeff Edwards, Partners at QHP. “We look forward to supporting the Lexitas team as they continue to build out their capabilities and provide best-in-class ophthalmic drug development services.” Additionally, Lexitas appointed Jeanne Hecht as Executive Chairwoman of the Board. Jeanne is a seasoned ophthalmology CRO industry executive having previously served as CEO of Ora, an ophthalmic CRO, where she expanded Ora’s international presence, introduced new lines of business, and furthered its goals along numerous strategic paths. About Lexitas Lexitas is a full-service ophthalmic biopharmaceutical solutions company focused on providing end-to-end eyecare support and exceptional service to biopharma and medical device clients globally. About QHP Capital QHP Capital, L.P. is the management company for NovaQuest Private Equity NQPE is a private equity firm that invests in lower middle market healthcare companies primarily in North America. The Firm invests in services and technology companies where a strategic partner and operating resources can accelerate growth, targeting companies in healthcare and pharmaceutical services industries. NQPE invests in the form of buyouts, growth equity, and recapitalizations.

Read More