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Vaccines Against COVID-19
Published in Hanadi Talal Ahmedah, Muhammad Riaz, Sagheer Ahmed, Marius Alexandru Moga, The Covid-19 Pandemic, 2023
Majid Khan, Muhammad Faheem, Najmur Rahman, Rizwan Ahmad, M. Zia-Ul-Haq, Muhammad Ria
When the safety and efficacy are shown in clinical trials, the vaccine manufacturer applies to national regulatory authorities (NRA) for registration or licensure of vaccine. This application is termed a biological license application (BLA) whereas, known as marketing authorization application in Europe and submitted to the submitted to the FDA in the US and European Medicines Agency (EMA) in Europe. The countries with a lack of existence of NRA, WHO provides licensure for vaccine production. Nevertheless, such vaccines still need licensure in a state where it has to be used.
Regulatory Challenges for Gene Delivery
Published in Yashwant Pathak, Gene Delivery, 2022
Vineet Mahajan, Shruti Saptarshi, Yashwant Pathak
USFDA via the Center for Biologics Evaluation and Research (CBER) and the National Institute of Health (NIH) via the Recombinant DNA Advisory Committee (RAC) are the apex bodies in the US that oversee regulatory affairs of gene therapy clinical trials and products.36,37 Gene therapy products intended for human use are classified and regulated as biological products38. The FDA regulatory system is comprised of several statutes, and guidance documents that companies have to comply with before initiating clinical trials for investigational gene therapy products. An approved biologics license (BLA) ensures the market authorization of gene therapy products in the US. Similar to the EU guidelines, gene therapy product trials have to meet strict guidelines in terms of quality, manufacturing process, non-clinical, and clinical aspects of the interventions. Additionally, there is an obligation for gene therapy products to adhere to good manufacturing regulations, good laboratory practice regulations, good clinical practice regulations, and good tissue practice regulations. It is also mandatory to provide proof of concept data and safety profile of the drug in animal models as per the set guidelines36. The FDA also oversees clinical trials of these products, unlike the EMA.
Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
FDA decides on the review designation for every application. However, an applicant may expressly request priority review as described in the Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics. It does not affect the length of the clinical trial period. FDA informs the applicant of a Priority Review designation within 60 days of the receipt of the original BLA, NDA, or efficacy supplement. Designation of a drug as “Priority” does not alter the scientific/medical standard for approval or the quality of evidence necessary.
Upstream cell culture process characterization and in-process control strategy development at pandemic speed
Published in mAbs, 2022
Jianlin Xu, Jianfa Ou, Kyle P. McHugh, Michael C. Borys, Anurag Khetan
In parallel to product and clinical development, CMC process development of mAbs for COVID-19 at pandemic speed is also required to meet urgent patient need. Process development for mAb manufacturing using Chinese hamster ovary (CHO) cells is relatively mature and has a long history of producing products with demonstrated safety to patients, high process yield, and suitable product quality requirements for human use. The entire product and process development from DNA for cell-line generation to biologics license application (BLA) submission usually takes 10 to 15 years.13,14 MAb process development is divided into upstream and downstream portions. The focus of this report is the upstream portion, which starts from vial thaw of a cell bank in a small shake flask and proceeds to seed culture expansion and final large-scale bioreactor production. Cell culture upstream process development includes two different stages: 1) the early-stage process development for investigational new drug (IND) application to the start of first-in-human (FIH) Phase 1 clinical trials, and 2) the late-stage development toward BLA submission for commercial use.
Long-acting ocular drug delivery technologies with clinical precedent
Published in Expert Opinion on Drug Delivery, 2022
Matthew N. O’Brien Laramy, Karthik Nagapudi
Approval from the US FDA may be granted under a biologics license application (BLA) or a new drug application (NDA), depending on the origin and type of active ingredient used in the product. Section 505 of the Federal Food, Drug, and Cosmetic Act describes three NDA filing pathways, depending on the amount of supporting data supplied by the applicant. For NDAs filed under Section 505(b)(1), the applicant collects and supplies all necessary data to support the approval. In contrast, NDAs filed under Section 505(b)(2) and 505(j) allow for abbreviated applications that leverage data not collected by the applicant and the Agency’s previous findings of safety and effectiveness. 505(b)(2) applications are differentiated from approved products and the FDA assigns a classification code to distinguish products by the unique or novel aspects of the application. 505(j) applications, or abbreviated NDAs (ANDAs), support the development of direct copy generic products.
Innovative approaches to biologic development on the trail of CT-P13: biosimilars, value-added medicines, and biobetters
Published in mAbs, 2021
HoUng Kim, Rieke Alten, Fraser Cummings, Silvio Danese, Geert D’Haens, Paul Emery, Subrata Ghosh, Cyrielle Gilletta de Saint Joseph, JongHyuk Lee, James O. Lindsay, Elena Nikiphorou, Ben Parker, Stefan Schreiber, Steven Simoens, Rene Westhovens, Ji Hoon Jeong, Laurent Peyrin-Biroulet
The EMA hybrid medicines pathway evaluates applications for “a generic medicine that is based on a reference medicine but has a different strength, a different route of administration or a slightly different indication from the reference medicine” that rely on data for both the reference and new products.73 In the US, applications under the 505(b)(2) pathway sometimes concern changes to approved drugs (for example, changes to dosage, strength, route of administration, or formulation) where the applicant relies on information from published literature or previous FDA findings.74 Until March 23, 2020, the 505(b)(2) pathway was also open to follow-on versions of biologics approved viaa New Drug Application – the regulatory process for biologic approval before the Biologics Price Competition and Innovation Act was adopted.75–77 After this date, biologics differing from approved products are unlikely to be considered under the biosimilar approval pathway by the FDA and will likely require a full BLA.75 While several recombinant protein therapeutics have been approved under the 505(b)(2) pathway in the past, some have been licensed as biosimilars in the EU,77 which could suggest that they do not offer clinically meaningful benefits for patients compared with their respective RPs.