• 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

    Convergence: First-year experience with Japan’s amended medical device rules

    Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) is providing priority review for certain innovative medical devices and has implemented a post approval change management process to allow for continuous improvement of medical devices.   The changes are part of the Amendment of the Pharmaceutical and Medical Device Act (PMD Act), which was implemented in September 2020. Officials from Japan’s Ministry of Health, Labour and Welfare (MHLW) and the PMDA provided a...
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    Key regulators featured in RAPS Convergence 2021 health authority forums

    On Monday, RAPS Convergence 2021 attendees will have the opportunity to hear from global regulators during three health authority forums. The presentations will highlight key topics and updates relevant to each regulatory body in hour-long sessions.   The session featuring Japan’s Pharmaceutical and Medical Devices Agency (PMDA) features four Japanese regulators, including session leader Tetsuya Kusakabe, PMDA’s international coordination officer, and Kanako Sasaki, de...
  • Regulatory NewsRegulatory News

    Convergence: Plenary probes how FDA, EMA will move forward

    During the opening plenary session for RAPS Convergence 2021, leading regulators from the US and EU moved beyond the now-obligatory discussion of lessons learned from the pandemic to a substantive review of how the agencies are really faring a year and a half into the public health emergency of the COVID-19 pandemic.   Speaking to an audience of regulatory experts in attendance at the virtual conference, Janet Woodcock, acting commissioner of the US Food and Drug Admin...
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    Industry calls for withdrawal of FDA electronic tracing guidance

    Pharmaceutical distributors urge the US Food and Drug Administration (FDA) to immediately withdraw its guidance setting parameters for establishing electronic systems to trace products through the supply chain. A trade association for distributors claims the agency overreached its statutory authority in seeking access to private electronic tracing information among trading partners.   The guidance, published in June, laid the groundwork for the data architecture system...
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    Kaleido warned; FDA not persuaded by 'medical food' claim

    The US Food and Drug Administration (FDA) recently issued a warning letter to Kaleido Biosciences for administering an investigational new COVID-19 treatment to patients without obtaining an investigational new drug (IND) application.   The company argued that the product under investigation was a food and not a drug, and therefore not subject to IND filing requirements. Yet the agency disagreed, saying that the endpoints used in the clinical trials went beyond ‘diet...
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    US to continue international inspection cooperation, says Marks

    Some of the US Food and Drug Administration’s (FDA’s) pandemic-related changes are likely here to stay, a top FDA official told Focus .   In the leadup to RAPS Convergence 2021, Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke with Focus about how CBER’s pandemic pivot accelerated a trend toward global cooperation, and how the changes stand to benefit patients and industry going forward.   Business as usual came to a halt...
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    FDA’s novel excipient pilot program opens for candidates

    The US Food and Drug Administration (FDA) has launched a new pilot program to review novel excipients for use in meeting unmet needs in formulating new drug products. The pilot has the full support of the pharmaceutical industry.   FDA called the program “a new pathway for manufacturers to obtain FDA review of certain novel excipients (inactive ingredients) prior to their use in drug formulations,” according to a 7 September announcement.   Excipients are inactiv...
  • RoundupsRoundups

    FDA Approvals Roundup: Trudhesa, Brukinsa, Keytruda

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approval Trudhesa nasal spray approved for migraine Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate nasal spray) has been approved for acute treatment of migraine with or without aura in adults. Other ergotamine formulations have been previously approved to treat migraine.   The approval was based on findings from the Phase 3, open-label STO...
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    FDA extends comment period on ICH Q12 guidance

    The US Food and Drug Administration (FDA) has reopened the comment period on its draft guidance implementing the International Council for Harmonization’s (ICH) Q12 guideline on postapproval chemistry, manufacturing and controls (CMC) changes for new and marketed pharmaceuticals and drug substances.   The additional time was requested by the Pharmaceutical Research and Manufacturers of America (PhRMA).   FDA announced in a pre-publication notice in the 8 Septembe...
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    FDA says Turkish OTC maker’s testing program earns failing grades

    The US Food and Drug Administration (FDA) criticized a Turkish manufacturer of over-the-counter drugs for failing to test incoming ingredients and to test products before releasing them to the market in a recent warning letter . The company was also warned for its lax approach to stability testing.   The 13 May letter to Istanbul-based Gulsah Uretim Kozmetik Sanayi Anonim Sirketi follows up on FDA’s review of a 23 April response to request for records. The firm is the...
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    News in Focus: PDUFA VII is a step in the right direction for the rare disease community

    Craig Martin is chief executive officer of Global Genes, a nonprofit organization whose mission is to increase public awareness for rare disease and globally connect, empower and educate individuals and rare disease foundations in person, print and online. More than 400 million people worldwide are affected by 7,000+ rare diseases. In the U.S., around 1 in 10 people (more than 33 million Americans) have a rare condition. More than half of rare disease patients are chi...