FDA advisors reject one Daiichi Sankyo cancer drug, back another

pharmaphorum | May 15, 2019

Daiichi Sankyo had a day of mixed fortunes after advisers to the US regulator voted against approval for its leukaemia drug quizartinib, but recommended pexidartinib for a rare tumour. Experts from the FDA’s Oncologic Drugs Advisory Committee (ODAC) met to discuss clinical data from the two drugs, and made non-binding recommendations ahead of the decision date set for them.
While these are only recommendations, the FDA usually follows the advice of its advisory committees. The Japanese pharma is seeking approval for quizartinib to treat patients with relapsed/refractory FLT3-ITD acute myeloid leukaemia (AML). But the committee voted against approval by eight votes to three, unconvinced by the results from the phase 3 QuANTUM-R study. Committee members agreed with FDA reviewers who raised concerns that although the study hit its target of a statistically significant improvement in overall survival, the study design may have biased the results. This was because there was a much higher rate of stem cell transplants in the quizartinib arm, than in a chemotherapy control arm.

Spotlight

Pharmaceutical manufacturing is shedding its “poor cousin” image and gaining in importance relative to R&D and marketing. Decades of regulatory and industry standard practice in manufacturing are being reworked to help push operating efficiency in pharma closer to other “normal” industries such as semiconductors, industrial chemicals and even consumer packaged goods. Early reports from the field suggest that progress will require massive change across organisations beyond the factory floor. However, the potential improvements in efficiency and regulatory oversight are too great to ignore.

Spotlight

Pharmaceutical manufacturing is shedding its “poor cousin” image and gaining in importance relative to R&D and marketing. Decades of regulatory and industry standard practice in manufacturing are being reworked to help push operating efficiency in pharma closer to other “normal” industries such as semiconductors, industrial chemicals and even consumer packaged goods. Early reports from the field suggest that progress will require massive change across organisations beyond the factory floor. However, the potential improvements in efficiency and regulatory oversight are too great to ignore.

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Societal CDMO Selected To Provide Tech Transfer And Manufacturing Services To Support Clinical Development Of An Oral Solid Dose Product In Europe

Societal CDMO, Inc. | July 21, 2022

Societal CDMO, Inc. a contract development and manufacturing organization dedicated to solving complex formulation and manufacturing challenges primarily in small molecule therapeutic development, today announced that it has been selected to provide CDMO services to support the ongoing clinical development of a novel drug candidate in Europe. The compound is an oral solid dose anti-viral therapy product approved for the prevention and treatment of human immunodeficiency virus in select countries in Europe and Societal’s work is focused on supporting the expansion of the product’s indications. Under terms of the new agreement, Societal CDMO will execute appropriate technology transfer activities, followed by cGMP manufacture of clinical trial material to support the initiation and execution of Phase 2 studies of the drug candidate. This work will include production, packaging and labeling of both the active compound and matching placebo for the study. “This is a key new agreement for Societal CDMO as it is a clear demonstration of the company’s ability to capitalize on the increasing interest of drug developers in the onshoring and reshoring of contract manufacturing activities to the U.S. from other regions around the globe. We are proud to be trusted by our partner to execute the tech transfer, manufacturing and packaging services that will be necessary for the initiation of its planned Phase 2 study in Europe. We continue to be pleased with our progress in leveraging our end-to-end CDMO service offerings to grow our business and expand our client base.” David Enloe, chief executive officer of Societal CDMO About Societal CDMO Societal CDMO is a bi-coastal contract development and manufacturing organization with capabilities spanning pre-Investigational New Drug (IND) development to commercial manufacturing and packaging for a wide range of therapeutic dosage forms with a primary focus in the area of small molecules. With an expertise in solving complex manufacturing problems, Societal CDMO is a leading CDMO providing therapeutic development, end-to-end regulatory support, clinical and commercial manufacturing, aseptic fill/finish, lyophilization, packaging and logistics services to the global pharmaceutical market. In addition to our experience in handling DEA controlled substances and developing and manufacturing modified-release dosage forms, Societal CDMO has the expertise to deliver on our clients’ pharmaceutical development and manufacturing projects, regardless of complexity level. We do all of this in our best-in-class facilities, which total 145,000 square feet, in Gainesville, Georgia and San Diego, California.

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DiMe Releases Toolkits to Improve Sensor Data Integration and Power Better, Faster Global Healthcare and Research

Digital Medicine Society | July 18, 2022

Today, the Digital Medicine Society released the Sensor Data Integrations Toolkits, four comprehensive toolkits to guide data producers, processors, and consumers to use the influx of data from the increased use of wearables and digital sensing products at scale. These resources are based on the Sensor Data Integrations project, comprised of healthcare leaders from Amazon Web Services Oracle, the Moffitt Cancer Center, Takeda, the US Department of Veterans Affairs and more. These toolkits will help realize the promise of sensor generated data to drive better decisions, faster, to improve healthcare delivery and research. The project team will conduct a demo of the toolkits during a live launch event on July 18 at 10:30 am ET, featuring Micky Tripathi, the National Coordinator for Health Information Technology at the US Department of Health and Human Services. The surge of data from sensor technologies is far outpacing the industry's ability to collect, store, analyze, protect, and use this data effectively for patient care and research. The number of US patients using remote patient monitoring devices is expected to surpass 70 million by 2025. Between 2022 and 2028, the global market for wearable technology is expected to grow around 18.5 percent, reaching $380.5 billion. The number of unique digital endpoints being used in industry sponsored trials of new medical products increased by over 950% between October 2019 and May 2022. The number of sponsors using these products in medical product development increased from 12 to 96 in the same timespan. And while the ability to discern high-quality sensor data suitable for clinical decision making is increasing rapidly, the ability to access these data is constrained by a current dependence on individual point solutions. "Sensor generated data, captured during people's daily lives, offer the opportunity to redefine how we measure health and disease. This opportunity powers the possibility of using high quality, high resolution flows of data to reimagine our approach to healthcare and research, leveraging more complete information to improve individual clinical decisions, decisions about the effectiveness of new medical products, and broader policy and public health decisions. DiMe's new Sensor Data Integrations Toolkits provide action-oriented resources to help data producers, processors, and consumers come together to create a sensor data ecosystem suitable for scale." DiMe CEO Jennifer Goldsack AWS has contributed customer feedback and technical expertise to ensure the toolkit meets the needs of today's modern healthcare system. "Across every industry, we see companies trying to accelerate their path to the cloud," said Lita Sands, Head of Solutions Life Sciences at AWS. "Timelines are getting quicker while the data collected is increasing exponentially – there is a clear and significant need for how to effectively and securely collect and use this information at scale. DiMe's new toolkits are a lifeline to organizations working with sensor data. They offer a comprehensive starting point for data producers, processors, and consumers to help build an integrated pipeline to support better and faster decision making." The development of these toolkits builds on DiMe's previous pre-competitive initiatives, such as The Playbook, the essential guide for developing and deploying digital clinical measures to advance patient care, clinical research, and public health. The Sensor Data Integrations Toolkits go further to ensure that high quality sensor data can be used at scale to improve patient care and speed efficient medical product development. The DiMe community is currently working on additional projects related to taking digitally generated data to scale and will be releasing additional resources over the coming months. DiMe is not only committed to creating and disseminating new digital health approaches and tools, but also sharing user experiences with the broader community. We encourage Sensor Data Integrations Toolkits users to contribute to Dime's "Resources in Action" case study hub by sharing how you are using resources to further the safe, effective, equitable, and ethical use of digital medicine to redefine healthcare and improve human health. The leading organizations from across the global healthcare, research, and digital health innovation sectors that collaborated with DiMe to create these open-access resources are AWS, Elevance Health, Evidation, US Food and Drug Administration (FDA), Human First, Institute of Electrical and Electronics Engineers, Medable, Moffitt Cancer Center, Open mHealth, Oracle, Savvy, Takeda, and US Department of Veterans Affairs (VA). About the Digital Medicine Society DiMe is a global non-profit and the professional home for all members of the digital medicine community. Together, we tackle the toughest digital medicine challenges, develop clinical-quality resources on a technology timeline, and deliver these actionable resources to the field via open-source channels and educational programs. Join us to advance the ethical, effective, equitable, and safe use of digital medicine to redefine healthcare and improve lives.

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Biocytogen Expands Partnership with Merck KGaA, Darmstadt, Germany

Biocytogen and Merck KGaA, Darmstadt, Germany | August 26, 2022

Biocytogen Pharmaceuticals Co., Ltd. entered into a global licensing agreement with Merck KGaA, Darmstadt, Germany, for the use of Biocytogen’s RenMiceTM platform. Under the terms of the agreement, Merck KGaA, Darmstadt, Germany will have full access to Biocytogen’s RenMiceTM platform to discover and develop fully human antibody therapeutics for an unlimited number of drug targets. Merck KGaA, Darmstadt, Germany will be responsible for all clinical development, manufacturing, and commercialization, and will provide Biocytogen with development and regulatory milestone payments. The official agreement was reached after an initial evaluation period. Dr. Yuelei Shen, Founder, Chairman and CEO of Biocytogen, said, “This agreement provides Merck KGaA, Darmstadt, Germany with one of the most advanced fully human antibody discovery platforms. We are pleased that Merck KGaA, Darmstadt, Germany recognized the potential of our RenMiceTM platform to accelerate antibody development for novel and challenging targets.” About Biocytogen Biocytogen Pharmaceuticals (Beijing) Co., Ltd. is a global biotechnology company that drives the research and development of novel antibody-based therapeutics. The company has streamlined the drug development process through the use of RenMiceTM for fully human antibody discovery, in vivo drug efficacy screening platforms and strong clinical development expertise. Biocytogen’s RenMiceTM HiTS Platform aims to develop novel antibody drugs with first-in-class and/or best-in-class potential for more than 1000 targets, and has resulted in collaborations with dozens of partners worldwide. The company's pipeline includes 12 core products; two products are in phase II MRCTs and two products are in phase I. Headquartered in Beijing, Biocytogen has branches in Haimen Jiangsu, Shanghai, Boston, USA and Heidelberg, Germany. About Merck KGaA, Darmstadt, Germany Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and electronics. Around 60,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2021, Merck KGaA, Darmstadt, Germany generated sales of €19.7 billion in 66 countries. Scientific exploration and responsible entrepreneurship have been key to Merck KGaA, Darmstadt, Germany's technological and scientific advances. This is how Merck KGaA, Darmstadt, Germany has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck KGaA, Darmstadt, Germany holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Electronics.

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