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  • Regulatory NewsRegulatory News

    FDA Issues Form 483 for Alexion’s Rhode Island Site

    Rare disease drugmaker Alexion disclosed late Monday in an SEC filing that the US Food and Drug Administration (FDA) recently completed a routine current Good Manufacturing Practice (GMP) inspection of the company’s Smithfield, Rhode Island manufacturing facility and issued a Form 483 with three observations. “These observations are inspectional, and do not represent a final FDA determination of compliance. The observations pertain to: completion and closure of certain ...
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    European Regulatory Roundup: EMA to Revise Trial Guidance on Hemophilia, Crohn’s, Ulcerative Colitis, Diabetes and Bipolar Disorder (4 August 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Boom in Hemophilia R&D Prompts EMA to act to Prevent Enrollment Bottleneck The European Medicines Agency (EMA) is planning to revise its guideline on development of drugs to treat hemophilia to cut the risk of patient enrollment bottlenecks slowing progress. EMA began the process after becoming concerned that the minimum data requirements of its current guideline are i...
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    House Looks to Reform Tropical Disease Priority Review Voucher Program

    The House Energy and Commerce Committee on Wednesday passed by voice vote an amendment (to an unrelated bill on hospital preparedness for dangerous threats) that would significantly change the priority review voucher (PRV) program for tropical diseases. Background Since 2009, the US Food and Drug Administration (FDA) has issued nine PRVs to reward companies for developing new drugs to treat rare pediatric and tropical diseases with a voucher that can be sold on the op...
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    Researchers Urge Caution on Expanding Priority Review Voucher Programs

    As Congress looks to further expand current priority review voucher (PRV) programs (and even create entirely new ones), researchers are now cautioning that the worth of the vouchers could decline significantly and diminish incentives to develop drugs for neglected diseases. Background Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers to pharmaceutical and biotech companies developing drugs and biologics fo...
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    Regulators, Industry Weigh in on Proposed EU Orphan Regulation Changes

    As the number of orphan drug approvals continues to rise in the EU, the European Commission is now considering feedback from European governments, regulators and industry on how changes to five aspects of the Orphan Regulation may further incentivize the development of drugs for rare diseases. To date, the commission has authorized 117 orphan medicines, 82% of which consist of new active substances, according to a new report .  And while the number of products authorize...
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    Neurological Device Trials: FDA Offers Draft Guidance

    The US Food and Drug Administration (FDA) on Friday released draft guidance to assist sponsors who intend to submit an investigational device exemption (IDE) to conduct clinical trials on medical devices targeting neurological disease progression, including for Alzheimer’s disease, Parkinson’s disease or primary dystonia. “Medical devices intended to slow, stop, or reverse the effects of neurological disease face challenges with regard to collecting safety and efficacy d...
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    Sanofi Redeems $245M Priority Review Voucher for Type 2 Diabetes Treatment

    The US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for Sanofi’s investigational type 2 diabetes treatment on Monday and granted the use of another Priority Review Voucher (PRV), which speeds up FDA’s decision by four months. In redeeming the voucher, Sanofi had to pay a $2.7 million fee in addition to the standard new drug filing fee of $2.4 million. Retrophin sold the PRV to Sanofi last May for one of the highest amounts eve...
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    EMA Offers New Draft Guidance on Developing Alzheimer’s Treatments

    The European Medicines Agency (EMA) on Monday released new draft guidance on the development of Alzheimer’s disease treatments that would allow treatments to be evaluated in earlier disease stages before dementia sets in. The new guidance comes as EMA recognizes that the field of Alzheimer’s disease (AD) research and development “witnessed a recent paradigm shift in the diagnostic framework of AD which is now considered a continuum with a long-lasting presymptomatic phas...
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    Orphan Medicines in the EU: A 15-Year Review

    With the 15th anniversary of the adoption of the Orphan Regulation in the EU, the European Commission notes in a new report that this landmark regulation has incentivized the development, approval and marketing of more than 100 rare disease treatments, or orphan drugs. According to the European Commission’s fourth inventory of orphan medicines, released Friday, somewhere between 5,000 and 8,000 different rare diseases exist in the EU, impacting between 27 million and 36 ...
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    First-in-Class, Rare Disease Drug Approvals Dominated in 2015: A Breakdown from FDA

    Last year was a banner year for the US Food and Drug Administration (FDA) in terms of new drug approvals, with many of the new drugs coming to market to help rare disease patients and offer new first-in-class options. The total of 45 approvals was well ahead of FDA’s average of about 28 novel approvals between 2006 and 2014, and 36% of those new approvals were for first-in-class drugs while almost half (47%) were to treat rare diseases, or ailments that impact 200,000 or...
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    FDA Faces Sensitive Challenge With Review of DMD Drug

    The blanketing of snow on the East Coast this past weekend prompted the postponement of a high-profile US Food and Drug Administration (FDA) advisory committee hearing for a Duchene Muscular Dystrophy (DMD) drug that is stirring up public controversy and will eventually lead to an up or down decision testing FDA’s resilience to public pressure. The advisory committee meeting, which the Jett Foundation, a DMD advocacy group, said Tuesday will likely be rescheduled withi...
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    Experts Call for New Regulatory Pathways for Antibiotics

    Faced with the increasing threat of antimicrobial resistance (AMR), experts are calling for more incentives to boost investment in new antibiotics. At the FDA/CMS Summit in Washington, DC today, representatives from several companies developing new antibiotic treatments said that while recent initiatives such as the Generating Antibiotics Now Act (GAIN Act) have helped revitalize investment in antibiotic development, more needs to be done to encourage research and deve...