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    Regulatory Focus, July issue: Cell and gene therapy

    Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational tria...
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    Regulation of advanced therapy medicinal products in the EU

    This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies...
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    Cell and gene therapies: FDA official on COVID-19 impact

    Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US Food and Drug Administration (FDA) official discussed the impact of the coronavirus disease (COVID-19) pandemic on the agency’s work related to cell and gene therapies.   “Before the pandemic, we were seeing a huge escalation in the workload that we have to make adjustments for,” said Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the Center for Biologics Eval...
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    FDA releases compliance program for CDER, CDRH-led combination product inspections

    In a newly issued compliance program , the US Food and Drug Administration (FDA) explains its approach to inspections of Center for Drug Evaluation and Research (CDER)- and Center for Devices and Radiological Health (CDRH)-led combination products.   The 46-page document lays out FDA’s intended implementation of the compliance program; inspectional operations and reporting considerations; sampling and analytical testing expectations; regulatory and administrative stra...
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    FDA abandons proposal for devices referencing drugs

    The US Food and Drug Administration (FDA) on Thursday said it will abandon a proposed regulatory approach for medical devices referencing drugs (DRDs) that would have allowed devices to be authorized for new uses with already approved drugs when the drugmaker does not want to collaborate on the new use.   The decision comes after FDA held a public hearing to discuss the scientific, regulatory and legal challenges posed by the approach in November 2017. At the time, FDA...
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    FDA drafts guidance on emergency-use injector reliability

    The US Food and Drug Administration (FDA) this week issued draft guidance explaining how combination product developers can demonstrate that their emergency-use injectors will reliably deliver drugs as intended in a life-threatening emergency.   The guidance specifically applies to emergency-use injectors that are prefilled or co-packaged with emergency drugs or biologics, such as those to treat anaphylaxis, and is intended to expand on FDA’s 2013 guidance Technical ...
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    As Stem Cell Clinics Proliferate, E&C Leaders Seek to Hear FDA’s Plan

    A bipartisan group of representatives on the Energy & Commerce (E&C) committee sent a letter to US Food and Drug Administration (FDA) Acting Commissioner Ned Sharpless on Thursday seeking more details on the agency’s plan to slow the proliferation of stem cell clinics. While noting that FDA has agreed to exercise enforcement discretion for 36 months in a recent guidance, the letter also explains how CBER Director Peter Marks said in April that industry has shown “mo...
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    FDA Questions Arizona Stem Cell Company

    As part of efforts to crack down on companies selling unapproved stem cell products to treat a variety of diseases and conditions, the US Food and Drug Administration (FDA) on Thursday sent an untitled letter to Arizona-based R3 Stem Cell and its more than 50 affiliate centers or clinics. FDA raises concerns with R3 because its website makes unsubstantiated claims about the stem cell products, which FDA says appear to be human cell, tissue, or cellular or tissue-based p...
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    FDA to Step Up Stem Cell Enforcement, Look Into Pathway for Low-Risk Treatments

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks on Wednesday warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies.   Gottlieb and Marks also said the agency will look into new ways to “delineate an efficient development path” for low-risk stem cell therapies being developed by firms that have filed investigational n...
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    Gottlieb, Marks Detail Plans to Advance Development of Cell and Gene Therapies

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks on Tuesday detailed plans for the agency to keep pace with an expected influx of applications for cell and gene therapies over the coming years.   "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications,...
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    FDA Warns Cord Blood Marketer, Reiterates Enforcement Discretion

    The US Food and Drug Administration (FDA) on Thursday released a warning letter sent late last month to San Diego-based umbilical cord blood product producer Genetech for selling unapproved products. The warning letter comes as FDA and the Centers for Disease Control and Prevention have received numerous reports of safety issues including those involving microbial contamination and are aware of 12 patients who received Genetech products and subsequently became ill due t...
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    FDA Looks Ahead and Back on Orally Inhaled and Nasal Generic Drugs

    The US Food and Drug Administration (FDA) has come a long way when it comes to locally acting orally inhaled and nasal drug products (OINDPs) and in the next five years of generic drug user fees, the agency is looking at further progress on some unique challenges, according to a regulatory science report on OINDPs. Background FDA’s Office of Generic Drugs (OGD) explained how the performance of OINDPs has been notoriously difficult to characterize because of a lac...