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    FDA issues clinical pharmacology draft guidance for antibody-drug conjugates

    The US Food and Drug Administration (FDA) on Monday issued a draft guidance on clinical pharmacology considerations for sponsors interested in developing antibody-drug conjugates (ADC). There is currently no guidance addressing these drugs, said the agency.   The guidance provides recommendations for ADCs, which are defined as an antibody or antibody fragment conjugated to at least one payload molecule via a chemical linker for treating cancer. They are regulated as ...
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    Orphan drug experts discuss new book on developing rare disease treatments

    Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...
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    Pharma, device groups oppose FDA's planned drug to device transition

    Pharmaceutical industry trade groups and medical device groups objected to the US Food and Drug Administration’s (FDA) plans to reclassify some products, including imaging agents, currently regulated as drugs to devices, in implementing a recent court decision. The Association for Accessible Medicines (AAM) says that the agency’s implementation of the order is hasty and has no legal merit, while the Advanced Medical Technology Association (ADVAMED) pointed out that compani...
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    FDA announces FY 2022 GDUFA science and research priorities

    The US Food and Drug Administration’s (FDA) Office of Generic Drugs (OGD) has announced its science and research priorities for fiscal year (FY) 2022 to spur the development of complex generic drugs. The priorities were prompted by public feedback the agency received at a public workshop earlier this year.   The agency also announced the availability of fellowships and funding for research activities that are aligned with these priorities.   The workshop was held i...
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    The RACE is on: Opportunities in pediatric oncology product development

    Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...
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    October’s Regulatory Focus: Linguistic review, advanced therapies in Asia-Pacific, and more

    Feature articles during October included an in-depth look at the linguistic review process in Europe and  reviews of the regulatory pathways for herbal medicinal products, medical devices, and supplements in the EU and for advanced therapies in the Asian-Pacific region. Also included was an article on the challenges of data integrity remediation in cGMP facilities.   Linguistic review and herbal product regulation The linguistic review process for centrally authori...
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    EU regulation of herbal products, Part 1: Definitions, regulations, and documentation

    This article compares and contrasts the regulatory requirements for herbal medicinal products, herbal-based medical devices, and botanical food supplements in the EU. It provides an overview of the current framework, highlights areas of controversy, and presents recent regulatory developments. The article is the first of two on this topic. The current article presents the definitions, main regulations, and documentation requirements for placing the products on the market;...
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    For gene therapies, FDA drafts trial guidance, finalizes "sameness" for orphan exclusivity

      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
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    FDA proposes to refuse Intarcia’s exenatide combo product NDA —again

    The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has told Intarcia Therapeutics it is proposing to refuse a new drug application for their type 2 diabetes drug-device combination product ITCA 650.   In a notice posted in the Federal Register , the agency said Intarcia can request a hearing from FDA on the proposal, but if the hearing is waived or it is determined the reason for the hearing isn’t sufficient, FDA will move forwa...
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    PDUFA VII commitment letter: RWE, rare diseases see renewed attention

    The US Food and Drug Administration (FDA) on Monday released the commitment letter outlining performance goals and procedures for the upcoming reauthorization of the Prescription Drug User Fee Amendments for FYs 2023-2027 (PDUFA VII).   The agency also announced a public meeting on the reauthorization, scheduled for the afternoon of 28 September. Registration for the meeting, which will be held virtually, is free.   Many of the concepts and programs the agency di...
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    EFPIA, others question EC's proposed orphan and pediatric drug development updates

    An initiative by the European Commission (EC) to boost the development of pediatric drugs and medicines for people with rare diseases has received stakeholder feedback that in many cases expresses concern with the proposed upending of the status quo.   A 60-day period of public consultation for the initiative’s roadmap closed at the end of July; in all, 112 comments were received from individuals, non-governmental organizations (NGOs), public authorities, medical socie...
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    EMA adopts guideline on quality documentation for drug-device combination products

    The European Medicines Agency (EMA) has adopted a guideline on the quality information that manufacturers of combination products should submit in marketing authorization applications (MAA).   It also incorporates the recent revision of the EU’s Medical Devices Regulations (MDR) in requiring makers of these products to obtain the results of conformity testing of the device part from a notified body. The MDR regulation went into effect in May 2021. (RELATED: EMA Kicks...