RESEARCH, PHARMA TECH
Ionis Pharmaceuticals, Inc. | March 10, 2023
Ionis Pharmaceuticals, a leading RNA-targeted therapy firm, recently announced that the USFDA has accepted for review a new drug application (NDA) for eplontersen. Eplontersen is an investigational antisense medicine designed to reduce the production of transthyretin (TTR) protein and treat hereditary and non-hereditary forms of ATTR amyloidosis (ATTR).
The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA has declared that no review concerns were detected and made no new data requests. The FDA also stated that it does not intend to conduct a meeting of the advisory committee to discuss the application.
ATTR amyloidosis is a systemic disease caused by the accumulation of TTR protein in organs, leading to progressive organ damage and eventually death. Patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) experience ongoing debilitating nerve damage throughout their bodies, resulting in the progressive loss of motor function.
The NDA for eplontersen is based on results from the global Phase 3 NEURO-TTRansform study showcased at the International Symposium on Amyloidosis (ISA). In the study, eplontersen showcased a statistically significant and clinically meaningful change relative to the baseline for its co-primary and critical secondary endpoints compared to the external placebo group. In addition, Eplontersen also demonstrated a favorable safety and tolerability profile.
Ionis and AstraZeneca announced a strategic partnership to develop and commercialize eplontersen in December 2021. Eplontersen is being developed and commercialized jointly by both businesses in the United States and will be created and commercialized by AstraZeneca throughout the rest of the globe, except Latin America.
The FDA granted eplontersen Orphan Drug Designation in the United States in January 2022. Eplontersen is also being studied in the worldwide Phase 3 CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and deadly disorder leading to progressive heart failure and death within 3 to 5 years after disease start.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company headquartered in Carlsbad, California. Its unique antisense technology platform allows them to develop RNA-targeted therapies that modify the production of disease-causing proteins, leading to the development of treatments for rare genetic disorders, cardiovascular diseases, and neurodegenerative diseases. The company's most significant achievement is developing and commercializing Spinraza, the first approved treatment for spinal muscular atrophy. Ionis Pharmaceuticals has also developed drugs for other rare diseases, including transthyretin amyloidosis, Huntington's disease, and myotonic dystrophy type 1.
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BUSINESS INSIGHTS, PHARMACY MARKET
Shields Health Solutions | March 17, 2023
Shields Health Solutions (Shields), the premier specialty pharmacy accelerator in the country, announced that it has partnered with Nemours Children’s Health, Delaware, to collaborate in developing a specialty pharmacy program that will provide patients with complex, chronic conditions access to specialized pharmacy services designed to lower costs, expand treatment options, enhance medication management, and improve overall health.
Shields will provide Nemours Children’s Health new support services through its partnership to meet the unique needs of patients with complex chronic conditions. Now, specialty pharmacy liaisons will work with patients on-site in clinics, offices, and pharmacies, among other locations, guiding patients who fill their prescriptions with Nemours Children’s. These liaisons work with teams of experts to assist patients and families with a multitude of tasks, including health insurance prior authorizations or securing financial assistance to help overcome one of the significant barriers to care.
These expanded patient support services, combined with access to recently approved and newly available drugs, will give Nemours’ patients greater availability for onsite treatment options. Shields currently partners with nearly 80 health systems around the country and has helped their patients reduce co-pays, promptly receive medication delivery, often within two days, and improved medication adherence greater than 90 percent on average.
Nemours Children’s Health is one of the largest multistate pediatric health systems in the country and provides award-winning care to more than 38,000 specialty patients. The complete health system includes two free-standing children's hospitals and a network of more than 70 primary, specialty and urgent care practices. US News & World Report ranked Nemours Children’s Hospital, Delaware among the 10 best children’s hospitals in the Mid-Atlantic region and recognized Nemours Children’s Hospital, Florida for excellence in orthopedics and endocrinology.
“Nemours’ commitment to outstanding care for children with chronic diseases aligns with Shields core mission,” said Stephen Davis, Senior Director of Health System Strategy. “Specialty pharmacy services provide challenges for all patient populations, but pediatric patients have unique needs that require tremendous support. We are proud to provide that support for patients at Nemours Children’s Health and help them achieve the best possible health outcomes.”
“As Nemours Children’s Health works toward creating the healthiest generations of children, it is imperative to address social determinants of health including access to medications that are affordable, said,” said Robert J. Mullen, BS, PharmD, RPh, Vice President, Patient Operations, Delaware Valley, at Nemours Children’s Health, Delaware. “Specialty pharmacy services can cause many challenges for the complex patients that Nemours Children’s cares for on a daily basis. We are proud to be partnering with Shields Health Solutions to help ease the burden for families acquiring prescription medication for their children and in turn achieve better health outcomes.”
About Shields Health Solutions
Shields Health Solutions (Shields) is the premier specialty pharmacy accelerator in the country. The Shields Performance Platform, an integrated set of solutions, services and technology, is intentionally designed to elevate payer and drug access for specialty pharmacies, elevate health outcomes for complex patients, and elevate growth throughout the entire health system. As the foremost experts in the health system specialty pharmacy industry, Shields has a proven track record of success including access to over 80 percent of all limited distribution drugs (LDDs) and most (health insurance) payers in the nation; and a clinical model proven to lower total cost of care by 13%. In partnership with more than 70 health systems across the country through national-scale collaboration, Shields has a vested interest in delivering measurable clinical and financial results for health systems.
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BUSINESS INSIGHTS, PHARMA TECH
US Pharmacopeia | January 04, 2023
The United States Pharmacopeia (USP) has announced the launch of the USP Advanced Manufacturing Technology Lab in Richmond, Virginia, as a part of its launch of a suite of R&D analytical solutions. These analytical lab services will aid drug manufacturers in adopting advanced manufacturing technologies, such as pharmaceutical continuous manufacturing (PCM), to increase geographic diversity in pharmaceutical manufacturing and support medicine supply chain resilience.
The new R&D analytical solutions will include analytical lab services that take advantage of USP's in-house scientific expertise and cutting-edge facilities at the USP Advanced Manufacturing Technology Lab in Richmond, as well as its headquarters in Rockville, Maryland. These capabilities will be utilized to characterize materials and qualify and develop ways to help ensure the quality of PCM-based medicines.
PCM involves a continuous flow of materials through a process pipeline in which two or more unit operations are directly connected and the sequential transformation of those materials. As the input materials are fed in, the process output, such as an active pharmaceutical ingredient or drug product, is collected, and the complete procedure is carried out in a single facility.
USP's emphasis on R&D analytical solutions and PCM-specific quality assessments is part of a broader set of activities aimed at addressing the problems of PCM adoption and bolstering pharmaceuticals' supply chain resilience.
Consulting services provided by a USP company, Pharmatech Associates, Inc., can assist manufacturers with decision-making and problems associated with PCM adoption. Pharmatech Associates consulting services, which operate independently of USP's standards-setting processes, provide manufacturers interested in PCM and other advanced manufacturing technologies with technical, business, and regulatory strategies, workforce training, and equipment and process design.
About The U.S. Pharmacopeia (USP)
USP is a non-profit, independent scientific organization that provides safe, high-quality pharmaceuticals. The organization has strived to establish confidence in the world's drugs, dietary supplements, and foods for more than two centuries. It protects patient safety and enhances global health via rigorous science and the establishment of public quality standards.
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