• RoundupsRoundups

    FDA Approvals Roundup: Tarpeyo, Dartisla ODT, Yusimry

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA). New approvals Tarpeyo approved for reducing proteinuria in IgA nephropathy Calliditas Therapeutics’ Tarpeyo (budesonide) delayed release capsules were granted accelerated approval to reduce proteinuria in adults with primary immunoglobulin A (IgA) nephropathy, also known as Berger’s disease, who are at risk of rapid disease progression. Approval of Tarpeyo wa...
  • Regulatory NewsRegulatory News

    This week at FDA: Medtronic MiniMed problems; Device classification flurry

    Welcome to 2021’s last issue of This Week at FDA. This week saw a year-end flurry of device classifications; we are also sharing news of three noteworthy device warning letters and a citizen petition response that closes with FDA’s referring a firm to the Federal Trade Commission (FTC) for anticompetitive practices.    RAPS offices are closed 23 December – 2 January. Regulatory Focus will resume publication on 3 January. From our team to you, our valued readers, best...
  • Regulatory NewsRegulatory News

    Euro Roundup: EMA reflects on a decade of collaboration with HCPs, recommends Janssen COVID booster

    The European Medicines Agency (EMA) recently released a report that reflected on a decade of implementing a framework to better work with doctors, pharmacists, and nurses in the EU.   As of December 2021, there are now 38 eligible organizations working with the agency in a variety of practice areas. The framework is designed for EMA to access a network of independent experts in clinical practice who can advise on real-world experience with drug development, benefit-ris...
  • Regulatory NewsRegulatory News

    Recon: Califf's Senate hearing; Moderna, Amgen pull out of JPM

      Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Former FDA commissioners endorse Califf as head of agency, citing urgency of addressing omicron ( WaPo ) Califf goes to Congress ( POLITICO ) Moderna, Amgen among biotech heavyweights pulling out of JPM conference over Covid concerns ( STAT ) US FDA’s Woodcock Sees Clinical Trial Reform Coming, But ‘Not Wholly Optimistic’ ( Pink Sheet ) Pfizer says its Co...
  • Regulatory NewsRegulatory News

    This Week at FDA: Bardoxalone strikes out at adcomm; Updated device consensus standards

    Happy Friday – this week we’re juggling lots of end-of-year activity from the US Food and Drug Administration (FDA). Today and in the next week, look for our contributors to highlight several draft and final guidance documents and to provide analysis and background on industry’s reactions to other guidances whose comment periods have recently closed.   The Center for Drug Evaluation and Research (CDER) released its fall/winter 2021 regulatory science news update today....
  • Regulatory NewsRegulatory News

    EC report details EU medicine shortages, offers potential solutions

    A commission ordered by the EC Directorate-General for Health and Food Safety (DG SANTE) to conduct a study on medicine shortages in the EU and European Economic Area (EEA) has released a final report outlining the problem and its recommendations for stakeholders.   Documenting the scope of human medicine shortages in EU/EEA countries between 2004 and 2020, the study conducted by Technopolis Group, Milieu Law & Policy Consulting and Ecorys analyzed data from 22 differe...
  • Regulatory NewsRegulatory News

    FDA issues draft guidance on study designs using real-world data

    The US Food and Drug Administration (FDA) has issued draft guidance to industry on how real-world data (RWD) could be used in clinical trials and how it could be incorporated into the design of non-interventional studies.   The draft guidance , released Thursday, outlines the applicability of FDA’s investigational new drug application (IND) regulations to clinical study designs that use RWD and clarifies the agency’s expectations on new drug applications (NDAs) or bio...
  • Regulatory NewsRegulatory News

    FDA rolls out more guidance on 'N of 1' gene therapies

    Developers of individualized investigational antisense oligonucleotide (ASO) therapies for ultra-rare diseases received additional guidance from the US Food and Drug Administration (FDA). In two draft documents, the agency has provided new information for sponsor-investigators and for those overseeing manufacture of these so-called “N of 1” therapies for people with severely debilitating or life-threatening genetic disease.   "Progress in individualized medicines provi...
  • Regulatory NewsRegulatory News

    ICMRA, WHO take stock of regulatory flexibilities

    A global regulatory group is mapping out a strategy for regulatory alignment as countries around the world plan their responses in the face of the Omicron variant of SARS-CoV-2, the virus that causes COVID-19.   The International Coalition of Medicines Regulatory Authorities (ICMRA) met on the first two days of December to discuss a host of regulatory challenges. Thirteen World Health Organization (WHO) and EC officials joined representatives from ICMRA’s 24 health aut...
  • Regulatory NewsRegulatory News

    Exclusive accelerated approval drugs comprise ‘relatively small share’ of Medicare spending

    Policymakers looking to rein in Medicare spending should look beyond the lens of the accelerated approval pathway, as drugs that exclusively fall under this indication make up a small percentage of overall Medicare spending, according to a recent research letter written by Benjamin N. Rome, of the Program on Regulation, Therapeutics, And Law (PORTAL) research group at Harvard Medical School and Brigham & Women’s Hospital, and colleagues.   “Medicare is spending more an...
  • Regulatory NewsRegulatory News

    FDA offers draft guidance for registries as RWD

    The US Food and Drug Administration (FDA) released draft guidance on the use of registries in regulatory decision making, adding another piece to the puzzle of how the agency will consider real-world data (RWD) when considering a new drug indication or assessing post-approval requirements.   The draft guidance , which was published Monday, outlines considerations for sponsors and stakeholders when proposing to design a registry or use an existing registry to support r...
  • Feature ArticlesFeature Articles

    Recent FDA approvals of drugs and biologics for children

    This article provides a summary of drugs and biologic products that have recently become available for the pediatric population in the United States. It highlights prescription products for attention deficit hyperactivity disorder (ADHD), psoriasis, active polyarticular juvenile idiopathic arthritis (pJIA), active psoriatic arthritis, Ebola virus treatment, spinal muscular atrophy, and bladder dysfunction that became available in 2019, 2020, and 2021 after completion of pe...