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    Devices Referencing Drugs: Questions Raised Over Potential Regulatory Pathways

    The US Food and Drug Administration (FDA) on Thursday held a public hearing to look into the scientific, regulatory and legal challenges posed by devices referencing drugs (DRDs) and the agency's proposed approach to regulating such products. Specifically, DRDs are medical devices that reference an already-marketed drug when the drugmaker does not want to work with the device sponsor to pursue the new use. FDA says it sees three situations where DRDs could be proposed: ...
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    Experts Tell Senate Committee Current Gene Editing Regulations are Appropriate

    At a Senate Health, Education, Labor and Pensions (HELP) hearing in Washington, D.C. on Tuesday, experts explained that the current regulatory framework for gene editing in the US is appropriate and cautioned that an overly strict approach could drive research to other countries. Specifically, the hearing focused on CRISPR/Cas9, which is a gene editing tool that scientists can use to quickly and accurately edit DNA in a variety of applications. While there are curr...
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    Asia Regulatory Roundup: Australia’s TGA to Tighten Regulation of Autologous Cell Products, Ban Ads (31 October 2017)

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia. TGA to Tighten Regulation of Autologous Cell Products, Ban Advertisements The Therapeutic Goods Administration (TGA) is planning to tighten oversight of autologous cell and tissue products. TGA’s proposed changes will bring some therapies under the biologicals regulatory framework, ban direct-to-consumer advertising and generally move Australia more in line with the ...
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    EMA, European Commission Look to Foster Development of Advanced Therapies

    The European Medicines Agency (EMA) and European Commission on Friday published a joint action plan to further the development of new cell and gene therapies, as well as other advanced therapy medicinal products (ATMPs). The plan, which features the release of several new and revised guidelines comes as on the international level, a regular forum for dialogue has been established to share experiences on advanced therapies between EMA, the US Food and Drug Administr...
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    FDA Panel Votes Unanimously in Favor of Gene Therapy to Treat Rare Eye Disorder

    Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 t...
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    New California Law Requires Posted Notices of Non-FDA Approved Stem Cell Products

    California Gov. Jerry Brown this week signed into law a bill that requires health providers to post notices in their offices when they are administering stem cell treatments that have not been approved by the US Food and Drug Administration (FDA). The law is part of efforts to crack down on the use and sale of unapproved stem cell treatments, some of which are being hawked as cures for a range of diseases with few or no treatment options, like ALS, autism and spina...
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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...
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    'Historic Action': FDA Approves First CAR-T Therapy

    The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...
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    FDA to Unveil New Regulatory Framework for Stem Cell Therapies

    The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...
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    Do Drugs Approved Via Expedited Pathways Offer Greater Benefits? Study and Doctors Debate

    A recent study in Health Affairs suggests that drugs given an expedited review by the US Food and Drug Administration (FDA) offer greater health gains than drugs that receiving a conventional review. But experts caution that the study might only show incremental benefits. The study, conducted by Peter Neumann, director of the Center for the Evaluation of Value and Risk in Health and three of his colleagues at Tufts Medical Center, compared the change in quality-adjuste...
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    FDA: US Clinical Research Using Three-Parent Baby Technique is Illegal

    The US Food and Drug Administration (FDA) said Friday that it is illegal to perform research in the US on a new type of technology that showed promise last year in helping a woman who had multiple miscarriages because of a genetic disorder to give birth to a baby boy in Mexico without that disorder. The technology, known as mitochondrial replacement technology (MRT), deals with mitochondrial DNA passed down only from mother to child. In September 2016, MRT was used to...
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    FDA Panel Votes Unanimously in Favor of First CAR-T Cancer Therapy

    The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy. Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the effi...