FDA promises elevated new drug approvals, but warns of potential pitfalls

fiercepharma | July 10, 2019

The FDA has approved 14 new drugs so far this year, lagging behind the 20 approvals it had charted by this time last year. Is it a sign that the FDA is becoming more cautious in the wake of Scott Gottlieb’s departure? After all, it was under his tutelage that the agency churned out a record 56 green lights in 2018. Don’t worry, biopharma industry: The FDA's still committed to accelerated drug development and approvals. That was the message from the director of the FDA’s Center for Drug Evaluation and Research, Janet Woodcock, M.D., who appeared Tuesday at the SVB Leerink Therapeutics Day in Boston. Woodcock said the “current elevated numbers” of new approvals would stay steady or even increase, according to a report SVB Leerink sent to clients today. “In fact, Dr. Woodcock believes that the drug industry is just beginning to see the fruits of the human genome sequencing and expects more and more approvals in future years,” the report said. During the SVB Leerink event, Woodcock said the FDA has been adapting to pharma's shift toward developing therapies for serious, difficult-to-treat diseases. “The FDA has heard from patients with these diseases that they have a willingness to assume a higher risk of uncertain efficacy and toxicity from investigational drugs and the agency has incorporated this into their review and approval decisions," the report said.

Spotlight

Alaska pharmacist Michelle Vaughn shares her story of pharmacists providing patient care. Pharmacists are health care providers, but they are not recognized as such under federal law. Provider status legislation would recognize pharmacists as health care providers, so patients will have improved access to pharmacists' services.

Spotlight

Alaska pharmacist Michelle Vaughn shares her story of pharmacists providing patient care. Pharmacists are health care providers, but they are not recognized as such under federal law. Provider status legislation would recognize pharmacists as health care providers, so patients will have improved access to pharmacists' services.

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PHARMACY MARKET

GluCare.Health Partners with Novo Nordisk for Innovative Weight Management Patient Program

Glucare.Health and NovoNordisk: | June 09, 2022

GluCare.Health, one of the Middle East's leading health tech startups, has partnered with global pharmaceutical company Novo Nordisk, market leader in Diabetes & Obesity, to introduce a blueprint for the future of healthcare. The partnership will see a 150-patient weight management pilot program that will combine new digital bio-markers, using wearable technology and devices for continuous data monitoring, with existing therapeutics. It introduces the Middle East's first value-based reimbursement model, and will be the first relationship of its kind for Novo Nordisk globally. This innovative partnership will put accountability for outcomes vs. costs on the provider, rather than on the insurers or patients. The 6-month program, which begins in September, is open to people with obesity who meet certain criteria. Patients will receive an unparalleled level of in-patient and data-driven remote care, with a multidisciplinary clinic team having continuous access to all patient data through an intelligent analytical platform. If patients do not lose at least 10% of their body weight by the end of the program, GluCare.Health and Novo Nordisk will cover all or a portion of the cost under the value-based reimbursement pilot. GluCare.Health is also committed to transparency when it comes to reporting results of the program. With one in three adults affected by obesity in the UAE[1], GluCare.Health's Co-Founder and Managing Director, Dr. Ihsan AlMarzooqi, says that the blueprint for care has the potential to not only save many lives but the UAE economy billions of dollars in costs and lost productivity. "When patients with chronic conditions are not supported properly, it can have a devastating impact on those individuals and their families. Additionally, the cost of treating the health consequences of obesity to the UAE economy alone is approximately $3.6 billion annually[2]. Traditional models of care remain woefully limited when it comes to outcomes. This partnership allows us to embed Novo Nordisk's advanced therapeutics into a companion platform like GluCare.Health, which enhances its efficacy dramatically." About Glucare.Health The first hybrid Digital Therapeutics (DTx)+Human vertically integrated diabetes platform of its kind in the world, GluCare.Health is reinventing metabolic care and transforming lives. Conceptualized in the UAE and opening the doors to its state-of-the-art 10,000 sq foot Dubai clinic in September 2020, it is the world's first healthcare provider to empower both clinicians and patients through Remote Continuous Data Monitoring as part of its standard model of care, an innovative and highly personalized 'continuous healthcare' approach that provides a comprehensive and real-time view of patients. As part of its unique model, GluCare is also an early adopter of - and the region's first clinic to use - Digital Therapeutics (DTx), combining it with wearable and smart technology, data analytics, unique in-clinic workflows, and a caring and connected expert care team. Applying technology as a humanizing force, GluCare.Health's team partners with patients and their families, giving them the tools, knowledge, and continuous support to live longer, healthier, and more fulfilling lives. About NovoNordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 49,300 people in 80 countries and markets its products in around 170 countries. For more information, visit Novo Nordisk UAE.

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BUSINESS INSIGHTS

ERS Genomics and Lepton Pharmaceuticals LTD Enter CRISPR/Cas9 License Agreement

ERS Genomics | July 27, 2022

ERS Genomics Limited is pleased to announce a new license agreement with Lepton Pharmaceuticals LTD. This is a non-exclusive licensing agreement granting Lepton access to the ERS CRISPR/Cas9 patent portfolio for certain applications. ERS, co-founded by Dr. Emmanuelle Charpentier, 2020 Nobel Prize winner for gene-editing, provides access to the foundational CRISPR/Cas9 intellectual property. Over 90 patents are held in over eighty countries. Lepton Pharmaceutical LTD is a biotechnology company developing a proprietary, breakthrough technology to substantially improve adaptive cell-mediated immune response. "We are very pleased to establish this relationship and license agreement with ERS Genomics. It will greatly assist us to develop our innovative Castling technology to significantly improve immune cells efficiency and longevity to fight cancer." Dr. Daniel Zurr, CEO of Lepton Eric Rhodes, CEO at ERS Genomics added: "Lepton's Castling technology has great potential to impact the field of immunotherapies and we are pleased to help enable Lepton in pursuing this exciting approach." The Castling technology is a proprietary, miRNA-based general technology platform, essentially consisting of the manipulation of miRNA expression to achieve enhanced efficacy and longevity (reduced exhaustion) of cell-based therapies. miRNAs are noncoding RNAs that each affects the expression of hundreds of coding genes. The Castling technology allows, in a single gene editing event in the immune cell (either allogeneic or autologous), to impact the expression of a multitude of genes in the immune cells, once such miRNAs penetrate the microenvironment of cancer cells. This technology aims to revolutionize the immune cell cancer therapy field. About ERS Genomics ERS Genomics is a biotechnology company based in Dublin, Ireland. The company was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property held by Dr. Emmanuelle Charpentier. Non-exclusive licenses are available for research and sale of products and services across multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products; companion animal and livestock health; production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology. About Lepton Pharmaceuticals Lepton Pharmaceuticals LTD. is based in Israel. Lepton focuses on subtle and precise silencing of coding RNA expression by small non-coding RNA molecules, working via the DROSHA/DICER/RISC pathways, small interfering RNA and microRNA (miRNA), to develop products and services to, ultimately, improve therapies for underserved diseases and improve and change patients' lives.

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BUSINESS INSIGHTS

Fusion Pharmaceuticals and Niowave Announce Actinium-225 Collaboration and Supply Agreement

Fusion Pharmaceuticals Inc. | June 13, 2022

Fusion Pharmaceuticals Inc. a clinical-stage oncology company focused on developing next-generation radio pharmaceuticals as precision medicines, and Niowave, Inc., a manufacturer of medical radioisotopes from radium and uranium, today announced that the companies have entered into a collaboration and supply agreement for the development, production, and supply of actinium-225. Under the agreement, Fusion will invest up to $5 million in Niowave to further develop their technology to increase current production capacity of actinium-225, and in return Fusion will have guaranteed access to a pre-determined percentage of Niowave's capacity of the resulting actinium-225, as well as preferred access to any excess supply produced. As part of the agreement, Fusion will also have an option to invest in future production of actinium-225 to scale with Fusion's needs. "As excitement for the tumor-killing potential of alpha-emitting radio pharmaceuticals increases, we intend to stay at the forefront of actinium development and supply to support our growing pipeline of targeted alpha therapies. We continue to prioritize manufacturing and access to actinium as a critical component of Fusion's platform, and our partnership with Niowave further strengthens and diversifies our supply chain as we advance multiple actinium-based radio pharmaceuticals in the clinic." Fusion Chief Executive Officer John Valliant, Ph.D "The Niowave team has worked hard to scale up our actinium-225 production to the millicurie level and this has allowed us to start working with oncology community partners," said Niowave Chief Executive Officer/Senior Scientist Terry Grimm, Ph.D. "We have been watching Fusion's progress in the development of their pipeline of targeted alpha therapies and we are very excited to partner with them on this journey." Fusion is developing actinium-based TATs leveraging the potency and precision offered by alpha particles. Actinium-225 decay gives off four alpha emissions in relatively rapid succession, maximizing the damage to the DNA of tumor cells, with a 10-day half life that allows for central manufacturing and distribution of products to clinical sites in a ready-to-use form. About Fusion Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines. Fusion connects alpha particle emitting isotopes to various targeting molecules in order to selectively deliver the alpha emitting payloads to tumors. Fusion's lead program, FPI-1434 targeting insulin-like growth factor 1 receptor, is currently in a Phase 1 clinical trial. The pipeline includes FPI-1966, targeting the fibroblast growth factor receptor 3 (FGFR3), advancing to a Phase 1 study following the investigational new drug (IND) clearance; and FPI-2059, a small molecule targeting neurotensin receptor 1 (NTSR1). In addition to a robust proprietary pipeline, Fusion has a collaboration with AstraZeneca to jointly develop novel targeted alpha therapies (TATs) and combination programs between Fusion's TATs and AstraZeneca's DNA Damage Repair Inhibitors (DDRis) and immuno-oncology agents. Fusion has also entered into a collaboration with Merck to evaluate FPI-1434 in combination with Merck's KEYTRUDA® (pembrolizumab) in patients with solid tumors expressing IGF-1R. Fusion and Hamilton, Ontario-based McMaster University are building a current Good Manufacturing Practice (GMP) compliant radiopharmaceutical manufacturing facility designed to support manufacturing of the Company's growing pipeline of TATs. About Niowave Niowave manufactures radioisotopes to cure cancer and save lives. Niowave builds and operates superconducting electron linear accelerators and is using that expertise to produce various radioisotopes for nuclear medicine. Niowave is a Cooperative Agreement partner with the National Nuclear Security Administration to develop a domestic supply of molybdenum-99 and currently produces yttrium-90 for use in cancer therapy. Niowave is licensed by the NRC to manufacture radioisotopes from uranium and radium and has partnerships with several universities and national laboratories focused on production and purification of radioisotopes for use in cancer therapy. By using radium targets and electron beams, Niowave is able to produce ultra pure and carrier-free actinium-225. Niowave's R&D facility is currently supplying actinium-225 samples to partners. Production-scale operations are ramping up at a second, FDA-compliant facility capable of meeting the growing actinium-225 market.

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