• Regulatory NewsRegulatory News

    FDA seeks new members for Patient Engagement Collaborative

    The US Food and Drug Administration is seeking eight new members for its Patient Engagement Collaborative (PEC), a group charged with gaining meaningful patient input into medical product development and regulatory decision making.   The 16-member panel includes patients, caregivers, and advocates who serve for 2 to 3 years. The panel is a joint effort between FDA and the Clinical Trials Transformation Initiative . Panel members are expected to participate in two to f...
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    Nouryant garners negative CHMP opinion for Parkinson's disease

    The human medicines committee of the European Medicines Agency (EMA) recommended two new medicines for approval during its July meeting; the committee also recommended four extensions of therapeutic indication and issued a negative opinion on one medicine.   Genzyme’s Nexviadyme (avalglucosidase alfa) was recommended by the Committee for Medicinal Products for Human Use (CHMP) to treat glycogen storage disease type II, also known as Pompe disease. Nexviadyme had receiv...
  • Feature ArticlesFeature Articles

    RWD, RWE, and improved patient outcomes: From FDA's Mini-Sentinel to real-world examples

    Uses of real-world data and evidence (RWD and RWE) have proliferated in recent years, driven by advances in data-gathering tools and methods and enhanced understanding of the value of such data and evidence. Under the 21st Century Cures Act of 2016, Congress codified RWD and RWE in the drug development and approval process. There are many opportunities for the use of RWD and RWE in the approval, labeling, clinical use, and marketing of medical products to enhance individu...
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    EC announces €120 million in Horizon Europe funding

    The European Commission (EC) has pruned the list of COVID-19-related research projects it plans to fund, announcing Thursday a short list of 11 projects that will receive €120 million in total.   Funding for the projects will come from Horizon Europe, a research and innovation program that launched in 2021 and is planned to sunset by 2027. The program supports “urgent research” into COVID-19 with a focus on the emerging threats of variants of SARS-CoV-2, according to...
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    FDA finalizes guidance on field alert reports

    The US Food and Drug Administration on Thursday finalized guidance on its expectations for applicants of new drug applications (NDAs) and abbreviated new drug applications (ANDAs) in submitting field alert reports (FARs) for potentially defective drugs. It contains only minor changes from the draft.   The final document supersedes a draft guidance that was issued in July 2018. (RELATED: Field Alert Report Submissions: FDA Offers Draft Q&A , Regulatory Focus 19 Jul...
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    CDRH official offers advice for smoother Q-submission meetings

    A policy analyst with the US Food and Drug Administration (FDA) walked through mechanisms the medical device industry can use to request pre-submission meetings, as well as best practices for ensuring smooth reviews.   Susannah Gilbert, Q-submission program lead with the division of regulatory programs at FDA’s Center for Devices and Radiological Health (CDRH), gave the updates at a 21 July webinar sponsored by the FDA’s Small Business Regulatory Education for Industry...
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    FDA seeks global shift to include pregnant women in medical research

    The COVID-19 pandemic has put a spotlight on the paradox surrounding the inclusion of pregnant and breastfeeding women in medical research. Even when these women are at higher risk for severe illness and hospitalization, they are unlikely to be included in clinical trials for medical products and vaccines. In a recent article, officials at the US Food and Drug Administration (FDA) signaled their commitment to work with industry and other regulators to support the inclus...
  • RoundupsRoundups

    FDA Approvals Roundup: Rezurock, Fexinidazole, Bylvay

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approvals Rezurock cleared for treating chronic graft-versus-host disease Kadmon’s Rezurock (belumosudil) has been approved for treating chronic graft-versus-host disease (GVHD) in adults and children aged 12 years or older after failure of previous systemic therapy.   Approval of the kinase inhibitor was based on efficacy findings from the randomized...
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    EMA begins rolling review of Sanofi Pasteur COVID vaccine

    The European Medicines Agency (EMA) has taken a fifth COVID-19 vaccine candidate into its rolling review scheme, evaluating data as they become available to speed a regulatory decision amid the ongoing public health emergency of the COVID-19 pandemic.   EMA’s decision to evaluate Sanofi Pasteur’s Vicprevtyn vaccine by rolling review is based on non-clinical data and early clinical studies “which suggest that the vaccine triggers the production of antibodies that target...
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    CBER targets four stem cell firms with untitled letters

    The US Food and Drug Administration’s Center for Biologics Evaluation and Research (FDA’s CBER) recently issued untitled letters to four companies for marketing unapproved stem cell products, indicating that despite the end of an enforcement grace period, companies are still resisting the agency’s efforts to get these products approved.   Effective June 1, companies are required to file biologics license applications for stem cell therapies that claim to treat a diseas...
  • ReconRecon

    Recon: $26B Opioids lawsuit deal imminent; Amylyx closes $135M ALS financing deal

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US How an Unproven Alzheimer’s Drug Got Approved ( NYT ) States and Cities Near Tentative $26 Billion Deal in Opioids Cases ( NYT ) Drug distributors settle New York opioid claims for up to $1.18B  ( Reuters ) Pharma CEOs, lobbyists showered Democrat with cash after his attempt to torpedo Pelosi’s drug pricing bill ( STAT ) Advocates urge Biden to name patent of...
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    Kineret under review by EMA for some COVID patients

    The European Medicines Agency has begun a new evaluation of an immunosuppressive drug to treat certain patients with COVID-19.   The agency announced Monday that it is reviewing an extension of indication for Kineret (anakinra, Swedish Orphan Biovitrum AB), to treat people with COVID-19 pneumonia at risk of developing severe respiratory failure. The human interleukin-1 (IL-1) antagonist was first authorized for use in the EU in 2002 and is currently authorized for th...