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    FDA guidance covers frequently asked CMC questions for generic drugs

    The US Food and Drug Administration’s Center for Drug Evaluation and Research (FDA’s CDER) on Monday issued a draft guidance that provides generic drug applicants with answers to commonly asked questions in the drug quality area. The new guidance is designed to provide an immediate response to such questions to reduce the number of controlled correspondence on identical topics.   The guidance, which is in a question-and-answer format, covers FDA’s policies on quality...
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    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...
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    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...
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    FDA’s revised MAPP outlines procedures for generic drug labeling changes

    The US Food and Drug Administration’s (FDA’s) Office of Generic Drugs recently issued a revised Manual of Policies and Procedures (MAPP) describing the agency’s internal procedures for handling generic drug labeling changes, including listing the responsibilities of the internal FDA staff responsible for managing these labeling changes.   The 16-page MAPP implements Section 10609 the Patient Protection and Affordable Care Act (PPACA) which was enacted in 2010 and add...
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    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....
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    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...
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    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 ) ...
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    EMA recommends bluebird bio's Skysona, seven other medicines

    The European Medicines Agency (EMA) issued a favorable opinion for bluebird bio’s Skysona (elivaldogene autotemcel), a gene therapy that treats early cerebral adrenoleukodystrophy (CALD). Seven other medicines also received positive recommendations from the agency’s Committee for Medicinal Products for Human Use (CHMP) during its May meeting.   Skysona’s status as an advanced therapy medicinal product (ATMP) means it was first assessed by EMA’s Committee for Advanced T...
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    FDA guidance spells out acceptance criteria for synthetic peptide ANDAs

    The US Food and Drug Administration (FDA) issued a final guidance on its criteria for accepting abbreviated new drug applications for synthetic peptide drugs of recombinant DNA (rDNA) origin.   The guidance is meant to spur the development of these complex drugs.  The agency also rejected industry calls to eliminate the impurity limit thresholds for accepting these submissions as abbreviated new drug applications (ANDAs).   The guidance, which finalizes 2017 draft ...
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    FDA releases batch of 21 product-specific guidances

    The US Food and Drug Administration (FDA) on Wednesday issued 13 new and eight revised draft product-specific guidances intended to facilitate the development of generic drugs.   The guidances are intended to foster generic competition by clarifying the agency’s expectations for the studies required to demonstrate that a generic drug is equivalent to a reference listed drug (RLD). FDA’s current count of product-specific guidances sits at 1,896 with this latest release....
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    FDA: Flexibility okay for gaps in gene therapy trials

    The US Food and Drug Administration (FDA) is not amenable to changing study endpoints or sample sizes for gene therapy clinical trials but is encouraging sponsors to fill in gaps in data collection with telemedicine and remote visits.   Wilson Bryan, director of the FDA’s Office of Tissues and Advanced Therapies (OTAT) in FDA’s Center for Biologics Evaluation and Research, told a 12 May meeting of the American Society of Gene and Cell Therapy that the pandemic is promp...
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    FDA welcomes alternative approaches to generic drug development

    Issuance of a product specific guidance should not “diminish innovation or creativity” in generic drug development, Robert Lionberger, director for the office of research and standards in the Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) Office of Generic Drugs, said at a 5 May webinar on the development of product specific guidance.   FDA officials said that the number of a product specific guidances (PSGs) issued by the Office of G...