• Regulatory NewsRegulatory News

    Nonclinical Studies for Enzyme Replacement Therapies: FDA Finalizes Guidance

    The US Food and Drug Administration (FDA) on Wednesday finalized guidance to help sponsors design and conduct nonclinical studies for developing investigational enzyme replacement therapy (ERT) products. ERT products, which generally involve exogenously supplying the missing or defective protein, can treat an array of disorders resulting from inherited defective genes (e.g., Gaucher disease, Fabry disease, Pompe disease and mucopolysaccharidoses), FDA explains. The n...
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    FDA Finalizes Guidance on Nonclinical Drug Development for Serious Hematologic Disorders

    The US Food and Drug Administration (FDA) on Thursday finalized guidance on the nonclinical studies drugmakers should conduct when developing products to treat severely debilitating or life-threatening hematologic disorders (SDLTHDs).   FDA classifies SDLTHDs as hematologic conditions that cause shorter life expectancy or greatly diminished quality of life even with available treatments.   FDA notes that the guidance does not apply to hematologic cancers and covers...
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    FDA Offers ICH S11 Guidance for Comment as Shutdown Threatens Further Guidance

    The US Food and Drug Administration (FDA) on Tuesday issued draft guidance, developed by the International Council for Harmonisation (ICH), on the standards for nonclinical safety studies in the development of pediatric medicines. The 38-page guidance should be consulted for study design, and early consultation is recommended for consideration of nonclinical support for pediatric medicine development. The guideline lays out advice on determining the need for additional ...
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    FDA Finalizes Testicular Toxicity Guidance

    The US Food and Drug Administration (FDA) on Wednesday finalized guidance for drugmakers on identifying and evaluating early signals that could indicate whether a drug might have adverse effects on the testes and when clinical trials are necessary to assess those risks.   Specifically, the 14-page guidance lays out recommendations for identifying nonclinical signals that suggest risk of testicular toxicity; conducting nonclinical assessments to further evaluate the ris...
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    Nonclinical Recommendations for Radiopharmaceuticals: FDA Drafts Guidance

    The US Food and Drug Administration (FDA) on Tuesday released draft guidance on nonclinical study recommendations for microdose radiopharmaceutical diagnostic drugs. The 7-page draft seeks to help developers of these microdose radiopharmaceutical diagnostic drugs on the nonclinical studies recommended to support human clinical trials and marketing authorization. The draft specifically discusses how to refine nonclinical study recommendations for this class of dru...
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    Pharma Companies Take Issue With FDA Proposal to Overhaul Nonclinical Study Regulations

    A group of the world’s largest biopharmaceutical companies, including Pfizer, Novartis, Celgene, Bristol-Myers Squibb and more than 170 others have offered their critiques and are seeking further clarifications on the US Food and Drug Administration’s (FDA) proposed rule to amend the regulations for good laboratory practice (GLP) for nonclinical laboratory studies. The proposed rule, unveiled in August , would require a complete quality system approach for these safety ...
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    FDA Revises Guidance on Safety Testing of Drug Metabolites

    The US Food and Drug Administration (FDA) on Tuesday released revised guidance on recommendations to industry on when and how to identify and characterize drug metabolites whose nonclinical toxicity needs to be evaluated. The revisions mean the new guidance supersedes guidance from February 2008. The guidance has been revised to be in alignment with ICH guidance, known as M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authori...
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    FDA Proposes New Rule to Overhaul Regulations for Nonclinical Studies

    The US Food and Drug Administration (FDA) on Tuesday proposed to amend the regulations for good laboratory practice (GLP) for nonclinical laboratory studies to require a complete quality system approach for these safety and toxicity studies. The 142-page proposed rule outlines what’s referred to as a “GLP Quality System,” which will involve additional management responsibilities and standard operating procedures (SOPs), as well as a more nuanced approach for multi-site ...
  • FDA Releases Draft Guidance for Veterinary Trials

    The US Food and Drug Administration's Center for Veterinary Medicine (CVM) has released a draft guidance detailing best practices for setting active controls in animal studies used to demonstrate effectiveness of medicinal products in cats, dogs and horses. As with clinical trials, FDA said the health and general welfare of study subjects should be an active consideration when determining whether to use placebo controls or active controls, which are defined as being a "k...
  • Feature ArticlesFeature Articles

    Demystifying Electronic Data Standards for Clinical and Nonclinical Studies

    As part of the Prescription Drug User Fee Act IV ( PDUFA IV ) information technology commitments, the US Food and Drug Administration (FDA) is moving toward a fully electronic, standards-based submission and review environment. 1 FDA has issued a series of guidance documents to assist sponsors in providing regulatory submissions in electronic format. In the latest draft guidance, Providing Regulatory Submissions in Electronic Format-Standardized Study Data 2 , issu...
  • New Initiative Aims to Bridge Drug Development's 'Valley of Death'

    • 01 March 2012
    What can be done to help companies get through the so-called 'valley of death' in drug development? University Hospitals, a Cleveland-area health system, wants to find out, and $250 million fund with the aim of fostering candidates through to late-stage clinical testing . The valley of death refers to the period between when non-clinical testing ends and late-stage clinical testing begins. Many drug candidates wash out of the process at this stage, either due to lack of...
  • Merck Gets Warning Letter After Failing to Complete Postmarketing Safety Study

    Merck Sharp and Dohme Corp.'s global director of regulatory affairs received a warning letter on 17 February from the US Food and Drug Administration warning Merck that it had failed to complete an agreed-upon postmarketing study for its diabetes medications Januvia and Janumet. The products are now effectively misbranded under the Federal Food, Drug and Cosmetic Act (FD&C Act), and the company faces $250,000 in fines, with further fines accruing for continuing vi...