• Regulatory NewsRegulatory News

    Marks eyes gene therapy development pilot, sees uptick in successful RMAT requests

    Peter Marks, director of Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), attributes an increase in the amount of regenerative medicine advanced therapy (RMAT) requests approved for cell and gene therapies to sponsors “getting the hang” of the agency’s expectations for designation requests.   Marks spoke at the California Separation Science Society’s (CASSS) meeting on 8 June on cell and gene therapies. The meeting was h...
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    Cavazzoni: Pandemic-era tools, learnings are here to stay

    The US Food and Drug Administration’s (FDA) top drug official said the pandemic has prompted the agency to consider new ways of working going forward and has renewed calls for industry to embrace digital technologies and decentralized models for conducting clinical trials. The pandemic has also prompted the agency to re-evaluate its hiring practices, and to adopt the use of alternative tools in lieu of onsite inspections, which have become a permanent fixture in the agency...
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    Experts say FDA enforcement focus unchanged, use of alternative tools to grow

    The US Food and Drug Administration (FDA) will continue to focus on data integrity practices and take a hard look at OTC firms’ testing programs either through onsite inspections or using alternative inspection tools in fiscal year 2022, according to experts who spoke to Regulatory Focus on near-term enforcement trends.   In addition, industry should expect to continue fielding requests for electronic records as the agency’s use of this alternative inspection tool is...
  • RoundupsRoundups

    FDA Approvals Roundup: Evrysdi, Kymriah, Tibsovo

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New indications Evrysdi gets label extension for use in babies with spinal muscular atrophy Genentech’s Evrysdi ( risdiplam; powder for oral solution ) has been granted a new indication to include babies younger than 2 months with spinal muscular atrophy (SMA).   The approval is based on interim findings from the RAINBOWFISH study in newborns demonstrat...
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    FDA and EMA mostly align on GCP inspection deficiencies, study finds

    A study conducted by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) found that the agencies are identifying the same types of deficiencies in joint good clinical practice (GCP) inspections with respect to protocol compliance, documentation deviations, and human subject protections.   The paper follows a mutual transatlantic cooperation on GCPs between the regulators that went into effect on 1 September 2009. Under the program, regulators ...
  • Feature ArticlesFeature Articles

    FDA ‘high-risk’ foreign inspections: Post‒COVID-19 response to GMP challenges

    This article examines the US Food and Drug Administration’s (FDA) return to inspections that were put on hold during the ongoing COVID-19 pandemic. It outlines the agency’s oversight of drug and biological products during the pandemic and its plans moving forward. The authors summarize the agency’s guidance and policies, industry considerations, and recommendations to manufacturers preparing for their next FDA inspection.   Keywords FDA, inspections, quality, risk m...
  • RoundupsRoundups

    FDA Approvals Roundup: Vtama, Dupixent, Vidaza

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approval Vtama approved as topical, steroid-free treatment for plaque psoriasis in adults Dermavant’s VTAMA ( tapinarof; cream, 1% ) has been approved as a steroid-free, topical treatment for adults with mild-severe plaque psoriasis.   Approval of the aryl hydrocarbon receptor agonist was based on findings from the Phase 3, multicenter, double-blind, ...
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    FDA asks manufacturers to develop risk management plans to avert shortages

    The US Food and Drug Administration (FDA) has issued draft guidance that spells out which types of drugs and medical devices are vulnerable to shortages and should be subject to risk management plan (RMP) reporting. The guidance also recommends risk factors to consider when developing the content of the RMPs.     Under the Coronavirus Aid, Relief, and Economic Security Act (CARES Act), FDA was given new authorities to collect information from manufacturers in respond...
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    FDA explains plans to bolster cell and gene therapy approvals through wider messaging

    Updated 25 May 2022 to correct ASGCT's name. Wilson Bryan director of the US Food and Drug Administration’s Office of Tissues and Advanced Therapies (OTA) in the agency’s Center for Biologics Evaluation and Research, told a 19 May meeting  of the American Society of Gene and Cell Therapy (ASGCT) that the division is undertaking a series of internal measures to improve communications with sponsors and to widen its messaging.   These measures are being taken to com...
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    FDA updates guidance on evaluating out-of-specification results for drugs

    The US Food and Drug Administration (FDA) has revised its 16-year-old final guidance on procedures for reviewing out-of-specification (OOS) results in the laboratory, including the responsibilities of the analyst and supervisor in reporting these results.   FDA defines OOS results as “all test results that fall outside the specifications or acceptance criteria established in dug applications, drug master files (DMFs), official compendia, or by the manufacturer. The ter...
  • RoundupsRoundups

    FDA Approvals Roundup: Mounjaro and Radicava ORS

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approvals Mounjaro okayed as dual-targeted therapy for type 2 diabetes Eli Lilly’s Mounjaro ( tirzepatide; injection ) has been approved for improving blood sugar control in adults with type 2 diabetes, in combination with diet and exercise.   Mounjaro is a dual agonist for the glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polyp...
  • RoundupsRoundups

    FDA Approvals Roundup: Olumiant, Enhertu

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New indications Olumiant use extended for hospitalized COVID-19 patients needing oxygen support Eli Lilly’s Olumiant (baricitinib tablets ) has been granted a new indication for treating COVID-19 in hospitalized adults who require varying degrees of ventilation/oxygen support.   Olumiant has been available to treat COVID-19 in the US under emergency use ...