• Regulatory NewsRegulatory News

    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      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the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   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
  • Regulatory NewsRegulatory News

    Convergence: Regulatory considerations in advancing gene and cellular therapies

    When developing a regulatory submission for a gene or cellular therapy, it’s essential to keep the basics in mind, such as how to demonstrate the product’s identity, purity, and potency, Haroon Hashmi, PhD, told attendees at RAPS Convergence 2021.   “Even when we think about these complex technologies -- new emerging technologies -- certain regulatory principles always stay in place,” said Hashmi, senior vice president at Ziopharm Oncology, which has developed the Slee...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    USP, NIST and NIIMBL collaborate to boost viral vector quality standards

    A new public-private collaborative among the United States Pharmacopeia (USP), the US Department of Commerce’s National Institute of Standards and Technology (NIST) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) pools the expertise or the organizations to develop better measurement tools to assess the quality of adeno-associated virus (AAV), an increasingly important vector for gene therapy delivery.   According to a 27 June ann...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    EMA's PRAC weighs in on more vaccine side effects

    The European Medicines Agency (EMA) has weighed in on several rare adverse events for COVID-19 vaccines and one gene therapy for treating thalassemia in their latest Pharmacovigilance Risk Assessment Committee (PRAC) meeting.   At its July meeting, EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) found mRNA-based COVID-19 vaccines, like Pfizer’s Comirnaty and the Moderna vaccine marketed in the EU as Spikevax, carry a small risk of myocarditis and pericarditis....
  • Regulatory NewsRegulatory News

    FDA calls for new warning labels on hydroxyethyl starch products

    The US Food and Drug Administration (FDA) on Thursday says it is requiring new safety warnings for hydroxyethyl starch (HES) products in light of reports of death, kidney injury and excessive bleeding associated with these products.   HES treatments are used to stabilize patients experiencing severe blood loss. The announcement follows up on an FDA safety review of these products from randomized clinical trials, meta-analyses, and observational studies. Through this re...
  • Regulatory NewsRegulatory News

    FDA issues draft guidance for transdermal adhesion systems

    The US Food and Drug Administration has issued new draft guidance for clinical trials examining the adhesion performance of transdermal and topical delivery systems (collectively, TDS) for new drug applications.   Surface area is an important variable in determining the amount of drug delivered through a TDS; adhesion performance can affect both safety and effectiveness of TDS products because adhesion failures can result in reduced effectiveness caused by suboptimal d...
  • Regulatory NewsRegulatory News

    EMA consults on ICH S12 guideline, shares comments on PFDD reflection paper

    The European Medicines Agency (EMA) on Thursday opened a public consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution (BD) considerations for gene therapies.   The S12 guideline recently reached Step 2b of the ICH process after being endorsed at the ICH Assembly virtual meeting earlier this month. (RELATED: ICH reports ‘significant milestones’ reached on guidelines ranging from impurity testing to eCTD standa...
  • RAPS' LatestRAPS' Latest
    RAPS AnnouncementsRAPS Announcements

    Author offers advice for navigating combination products approval pathways

    With few comprehensive resources available covering combination products, RAPS’ recently released book, International Combination Products , by Jocelyn Jennings, MS, RAC (US, Drugs, Devices), fills a significant need for practical guidance on navigating the regulatory pathways for products combining multiple technologies. Prior to writing the book, “there was no place that I found that I could look to find all of the information I was interested in on different topics t...
  • ReconRecon

    Recon: ASCO 21 updates; Moderna goes for teen EUA

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Moderna says that its vaccine is effective for 12- to 17-year-olds and that it will seek F.D.A. authorization ( NYT ) ( NPR ) President Biden still hasn’t appointed a permanent FDA commissioner. That has big implications for upcoming drug approvals ( Fortune ) Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic ( Global Genes ) ...