• Regulatory NewsRegulatory News

    FDA to Step Up Stem Cell Enforcement, Look Into Pathway for Low-Risk Treatments

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks on Wednesday warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies.   Gottlieb and Marks also said the agency will look into new ways to “delineate an efficient development path” for low-risk stem cell therapies being developed by firms that have filed investigational n...
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    FDA Offers Guidance on Nicotine Replacement Therapies

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance providing recommendations to drugmakers looking to develop nicotine replacement therapy (NRT) products to help patients quit smoking.   The 19-page draft guidance comes after FDA held a public consultation on its approach to evaluating NRT products in November 2017 and a public hearing on the matter in January 2018.   FDA says the draft guidance “takes into consideration the feedback re...
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    CBER Guidance on Gene Therapies: What to Expect in 2019

    The US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) on Wednesday released plans for what guidance is to be expected in 2019, including six final guidance documents related to gene therapies. The gene therapy guidance documents ( draft versions were published in July ) the agency expects to finalize include: Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry Chemistry, Manufacturing, an...
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    510(k) Modernization: FDA Floats Need to Pursue New Authority

    Plans at the US Food and Drug Administration (FDA) to “modernize” the most commonly used pathway for new medical device clearances “might require new authority,” FDA leadership said on Monday. FDA Commissioner Scott Gottlieb and Jeff Shuren, director of FDA’s Center for Devices and Radiological Health (CDRH), issued a joint statement to announce 2018 progress on novel device approvals, pointing out that the breakthrough 510(k) is one of two submission types that was new...
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    Industry Calls for Metrics on CDRH’s Breakthrough Device Program

    A consensus of industry participants at a US Food and Drug Administration (FDA) webinar on Thursday pressed for metrics on the medical device priority review program run by FDA’s Center for Devices and Radiological Health (CDRH). The webinar led by Maureen Dreher, policy analyst at CDRH’s Office of Device Evaluation, underscored the need for additional transparency around the Breakthrough Device Program. This request was supported across several participants during the ...
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    FDA Finalizes Breakthrough Device Guidance, Outlines Safer Technologies Program

    The US Food and Drug Administration (FDA) on Tuesday finalized guidance detailing its breakthrough devices program and announced plans to bring safer medical device alternatives to market via a new safer technologies program (STeP).   “We continue to encourage device manufacturers to consider the breakthrough devices program for innovative, lifesaving devices, and, in the near future, our goal is to have STeP as an option for those devices with new advances and adaptat...
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    BIO Seeks Clarity on FDA’s Gene Therapy Draft Guidances

    Industry group BIO recently submitted comments on a series of new draft guidance documents on gene therapy released by the US Food and Drug Administration (FDA) in July. The six guidance documents focus on developing hemophilia, rare disease and retinal disorder gene therapies, and also include one on chemistry, manufacturing and control (CMC) information, one on long term follow-up observational studies collecting data on adverse events and one on the testing of retr...
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    FDA and Pentagon Forge Breakthrough Designation Agreement

    Following the Pentagon’s criticism of the US Food and Drug Administration’s (FDA) sluggishness in approving a French freeze-dried plasma product, which some have said other countries’ troops have used for years, FDA and the Department of Defense’s (DoD) Office of Health Affairs on Tuesday launched a joint program to prioritize the efficient development of such medical products. The framework for the program was established, via H.R.4374 , after DoD sought to crea...
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    FDA Approves New Gene Therapy Targeting Specific Mutation

    The US Food and Drug Administration (FDA) on Tuesday approved a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness. The approval comes two months and a week after an advisory committee of outside experts unanimously voted in favor of approving the treatment for the condition which affects about 6,000 worldwide. The price of the new gene therapy, which Leerink...
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    Resolving Gene Editing Technology's Ethical and Regulatory Challenges

    This article provides an overview of biomedical applications of gene editing technology, addresses ethical and regulatory challenges associated with its implementation for therapeutic development, and proposes approaches for overcoming these challenges. Introduction Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology is a genetic "editing" tool aimed at altering DNA sequences and modifying gene function. It offers promising opportunities for ...
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    FDA, CMS: Second Parallel Review Decision Ever for NGS Test

    The US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS) on Thursday announced joint approval and coverage decisions for Foundation Medicine's FoundationOne CDx next generation sequencing (NGS) based test. The test was reviewed under FDA and CMS' parallel review program, and is only the second test to be approved and cleared under the program since its inception in 2011, the first being Exact Sciences' Cologuard colorectal cance...
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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...