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
Businesswire | March 27, 2023
Massive Bio, Inc., a private AI-enabled oncology startup that provides virtual and in-person concierge services for cancer patients, and NeoGenomics, Inc. a leading provider of cancer-focused genetic testing services and global oncology contract research services, today announced a collaboration with the goal of accelerating the development of new cancer therapies and ultimately improving the lives of millions of cancer patients around the world. NeoGenomics will identify patients in real time who may be eligible for clinical trials based on biomarker status. Following initial contact and outreach provided directly from NeoGenomics to the treating physician, Massive Bio will help obtain patient consent and expediate additional screening and potential enrollment. This partnership will help to quickly identify patients eligible for clinical trials and help patients and providers make an informed decision regarding their potential treatment avenue.
By combining their respective strengths in biomarker testing, data analysis, machine learning, and biomarker and genomic profiling, the collaboration between NeoGenomics and Massive Bio have achieved a significant milestone in the oncology industry.
"Our mission at Massive Bio is to provide cancer patients with the best possible care and treatment options,” said Selin Kurnaz, PhD, CEO and co-founder of Massive Bio. “By partnering with NeoGenomics, a leading player in the cancer diagnostics industry that shares Massive Bio’s commitment to advancing cancer research and improving patient outcomes, we can leverage their expertise in oncology diagnostics to accelerate the identification of patients who may be eligible for clinical trials."
“NeoGenomics’ advanced diagnostic tools and U.S. footprint, combined with Massive Bio's AI capabilities and concierge services in oncology, will enable us to match patients to clinical trials faster and more efficiently, resulting in improved outcomes and reduced costs,” said Vishal Sikri, President of the Advanced Diagnostics Division of NeoGenomics. “We are thrilled to partner with Massive Bio to advance precision medicine and improve the delivery of healthcare services to patients, pharmaceutical partners, and healthcare providers."
Dr. Arturo Loaiza-Bonilla, MD, co-founder and Chief Medical Officer of Massive Bio, added, "This collaboration between NeoGenomics and Massive Bio will enable us to provide patients with personalized care and support throughout their cancer journey, and move the needle forward in precision oncology and research."
AboutMassive Bio
Massive Bio empowers cancer patients to find their best treatment options, using artificial intelligence to improve equitable access and precision targeting for clinical trial matching, drug matching, and drug development. Massive Bio combines its best-in-class AI platform with technology-enabled services to remove barriers in clinical trial enrollment, value-based oncology decisions, and data-driven cancer treatment. The company serves more than two dozen pharmaceutical companies, contract research organizations, and hospital networks, and has been awarded an SBIR contract by the National Cancer Institute. Massive Bio was founded in 2015 by clinical, technology, and M&A executives, and has a global presence with nearly 100 people in 12 countries.
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BUSINESS INSIGHTS, PHARMACY MARKET
West Pharmaceutical Services, Inc. | February 01, 2023
On January 31, 2023, West Pharmaceutical Services, Inc., a global pioneer in novel injectable drug administration solutions, announced its participation in Pharmapack Europe 2023 in Paris, France, to demonstrate its leadership and insights on industry packaging and containment trends and challenges.
With the introduction of three new products, the company expands its innovations for its patients and customers worldwide - Daikyo Crystal Zenith® 2.25mL Insert Needle Syringe System, West Ready Pack™ with Corning Valor® RTU Vials using Stevanato Group's EZ-fill® SG EZ-fill® technology and FluroTec® 5-10mL Cartridge Plunger.
West Pharmaceutical Services Vice President and General Manager, Biologics Li Chen commented, "This year, we are introducing three new product solutions at Pharmapack." He added, "With these innovations, we are addressing the growing market needs for today's complex and sensitive molecules, including large-volume delivery and a complete vial containment solution that can protect and scale from development through commercialization."
(Source – PR Newswire)
Meanwhile, West's participation in this year's conference includes the following
Learning Lab - How Market Trends Drive Innovation and Enable the Pharmaceutical Pipeline on Wednesday, February 1, 2023
Panel Session - Innovation of Novel Medicines and Trends in Demand for Injectable Home and Self-Administration on Wednesday, February 1, 2023
Innovation Tours - West Ready Pack™ with Corning Valor® RTU Vials using Stevanato Group's EZ-fill® Technology on Wednesday, February 1, 2023
Daikyo Crystal Zenith® 2.25mL Insert Needle Syringe System on Thursday, February 2, 2023
About West Pharmaceutical Services, Inc.
Founded in 1923, West Pharmaceutical Services, Inc. is a market leader in developing and delivering innovative, high-quality injectable solutions and services. As a trusted partner to both established and emergent drug producers, it contributes to the safe, effective containment and distribution of life-saving and life-enhancing medications to patients. West Pharmaceutical supports its clients by supplying over 45 billion components and devices annually, with roughly 10,000 team members spread across 50 facilities globally. The company marks its 100th anniversary of growth and innovation in 2023, as well as the essential role it continues to play in healthcare and the patient experience. Headquartered in Exton, Pennsylvania, it had generated $2.83 billion in net sales for the fiscal year 2021.
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