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  • Regulatory NewsRegulatory News

    First in vitro diagnostic for early Alzheimer’s detection gets FDA blessing

    The US Food and Drug Administration (FDA) has authorized the first in vitro diagnostic to help diagnose early Alzheimer’s disease. The agency said the test minimizes radiation risks to patients who otherwise would need to be tested with positron emission tomography (PET) scans.   On 4 May, FDA announced it has given the green light to Fujirebio Diagnostics’ de novo application for its Lumipulse G β-Amyloid Ratio (1-42/1-40) test, which is intended for patients ages...
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    RAPS' LatestRAPS' Latest

    Orphan drug experts discuss new book on developing rare disease treatments

    Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...
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    This Week at FDA: New proposed rules, Biocon's Form 483, and more

    We're trying something new at Regulatory Focus , with the goal of gathering the week’s news from (and about) FDA. We'll be covering agency activities and enforcement actions, filings in the Federal Register , new guidances, legislative updates, and anything else we come across – all in one place. We hope you will find it to be your one-stop-shop for FDA updates. What do you think? We’d be happy to hear your feedback at news@raps.org. News from this week One of t...
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    US regulations for regenerative medicine advanced therapies

    This article examines US regulations and guidance documents for regenerative medicine advanced therapies (RMATs). The author describes the field of regenerative medicine, noting that it is expanding at an accelerated pace, and outlines some of the common terms associated with it. He also addresses the application process for these therapies, accelerated regulatory pathways, market access, and the outlook for RMATs. The author cautions that, as exciting as these therapies a...
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    Update on RMAT designations

    This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   Introduction Section 3033 of the 21st Century Cures Act, titl...
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    Two Gene Therapy Approvals Headline CBER’s FY 2018 Report

    The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) on Wednesday published its Fiscal Year 2018 report, highlighting two gene therapy approvals, as well as the approvals of a hepatitis B vaccine, a vaccine to prevent shingles and a flu vaccine for children as young as six months. Both of the gene therapy approvals occurred at the very beginning of the previous fiscal year, with CBER signing off on Spark Therapeutics’ Luxturna...
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    Drugmaker Petitions FDA to Revoke Sublocade Orphan Designation

    A citizen petition filed last week by law firm Goodwin Procter on behalf of Pennsylvania-based drugmaker Braeburn calls on the US Food and Drug Administration (FDA) to revoke the orphan designation granted to Indivior’s opioid use disorder treatment Sublocade (buprenorphine extended-release injection).   “Sublocade is not now, nor was it ever a bona fide orphan drug, particularly since more than two million Americans currently are afflicted by opioid addiction,” the pe...
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    European Commission Consults on Orphan Drug Applications

    The European Commission on Tuesday launched a targeted consultation to provide input on a revision of its guideline on the content and format of applications for orphan drug designation, as well as the transfer of designations between sponsors.   "The comments received will be taken into account by the European Commission in the finalization of the guideline on the format and content of application for designation as orphan medicinal products and on the transfer of des...
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    FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements

    The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions. Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of th...
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    FDA Unveils New Regenerative Medicine Framework

    The US Food and Drug Administration (FDA) on Thursday launched a new policy framework for regenerative medicine, building off a previous framework from 2005, as part of efforts to bring new cell, stem cell and tissue products to patients as efficiently as possible while managing the proliferation of unscrupulous actors hawking unproven therapies. FDA’s announcement included the release of two new draft guidance documents – one on ways to expedite approvals for regen...
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    EMA Backs Six Drugs, One Biosimilar for EU Approval

    The European Medicines Agency (EMA) on Friday said that its Committee for Medicinal Products for Human Use (CHMP) is recommending six new drugs, a biosimilar and three generic medicines for EU approval. The six medicines the agency is recommending are: Dr. Falk Pharma's Jorveza (budesonide) to treat eosinophilic esophagitis, a rare inflammatory condition of the esophagus. Jorveza was granted an accelerated assessment and orphan designation; Merck Sharp & Dohme's Pr...
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    FDA Finalizes Combination Product Classification Guidance

    The US Food and Drug Administration (FDA) on Tuesday issued its final guidance on classifying combination products as drugs, biologics or medical devices. The guidance finalizes and combines two 2011 draft guidances, Classification of Products as Drugs and Devices & Additional Product Classification Issues and Interpretation of the Term 'Chemical Action' in the Definition of Device Under Section 201(h) of the Federal Food, Drug and Cosmetic Act , and details how F...