• Regulatory NewsRegulatory News

    EU Regulatory Roundup: UK Sets up Logistics Hub in Belgium to Support Post-Brexit Drug Shipments

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU   regulatory news.   UK Sets up Logistics Hub in Belgium to Support Post-Brexit Drug Shipments   The United Kingdom has created a logistics hub and bought tickets from ferry operators to mitigate the risk that a no-deal Brexit will disrupt the supply of drugs and medical devices.   In the event of a no-deal Brexit, preferred shipping routes between Dover, Calais and other towns and ...
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    FDA Approves First Treatment for a Form of Batten Disease

    The US Food and Drug Administration (FDA) on Thursday approved the first-ever treatment for a form of the rare pediatric Batten disease, bringing Biomarin its second lucrative priority review voucher (PRV). FDA approved Brineura (cerliponase alfa) to slow the loss of walking ability in symptomatic pediatric patients three years old and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. ...
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    FDA Panel Votes Against Approval for Sarepta DMD Drug

    Following a contentious debate pitting young boys with the terminal illness Duchene Muscular Dystrophy (DMD) against the US Food and Drug Administration (FDA), an advisory panel of outside experts on Monday voted against the approval of Sarepta Therapeutics’ controversial investigational drug eteplirsen to treat DMD. The agency’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7-3 against approval, with three abstaining, and 7-6 against the accelerat...
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    First Pediatric Priority Review Voucher Goes up for Sale, Fetching $67M

    As far as regulatory incentives go, some are pursued more than others. The US Food and Drug Administration's (FDA) orphan drug exclusivity provisions, for example, have brought about a renaissance of therapies meant to treat patients suffering from rare diseases and conditions. But on the other side of the coin are incentives not often sought out by companies. And perhaps no incentive maintained by FDA is used less than its priority review vouchers. Background: Vouchers...