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    EMA Consults on Regulatory Requirements for Chronic Liver Disease Drugs

    The European Medicines Agency (EMA) on Thursday launched a public consultation to gather input on a future reflection paper discussing the regulatory requirements for developing drugs to treat chronic non-infectious liver diseases. According to the agency, there is an unmet medical need for drugs to treat chronic non-infectious liver diseases such as non-alcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). And wh...
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    European Commission and EMA Offer Guidance on Brexit for Pharma Companies

    As marketing authorisation holders (MAHs) based in the UK begin to form plans to transfer their marketing authorizations (MAs) to holders established in the EU, Norway, Iceland and Liechtenstein, the European Medicines Agency (EMA) and European Commission on Wednesday released guidance to prepare industry for the UK’s withdrawal from the EU. The question and answer document is the first in a series of guidances and follows a notice sent earlier this month to ...
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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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    TGA to Move Forward With Orphan Drug Revamp

    Australia's pharmaceutical industry is "broadly supportive" of proposed changes to the country's orphan drug program, the Therapeutic Goods Administration (TGA) says. On Tuesday, TGA released feedback it received in response to a 2016 consultation on the proposed changes, which include raising the population threshold for rare diseases, taking the seriousness of the condition into account when making designations and requiring sponsors to demonstrate significant benefit ...
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    GAO: Drugmakers Want More Clarity on Antibiotic Incentives

    Despite some improvements, drugmakers say the US Food and Drug Administration (FDA) has not fully clarified its expectations for developing new antibiotic drugs or detailed how to access new incentives, according to a new report by the Government Accountability Office (GAO). Specifically, GAO says that FDA has issued 14 updated or new guidances focused on antibiotics since the Generating Antibiotic Incentives Now (GAIN) Act was passed as part of the Food and Drug Admi...
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    Sen. Grassley Opens Inquiry Into Orphan Drug Act

    Sen. Charles Grassley (R-IA) on Friday confirmed to Focus that he is gathering more information and discussing with his staff and interested parties a possible inquiry into the Orphan Drug Act ’s abuses leading to high drug prices. “Based on the reporting from Kaiser Health News [KHN] about how the orphan drug provisions appear to be stretched beyond their original intent, and the strong consumer concern about high drug prices, I'm interested in learning whether...
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    European Commission Clears up Questions on Orphan Drug Regulation

    Following a public consultation in November 2015, the European Commission (EC) has released a new communication intended to clarify some lingering questions about the EU's Orphan Regulation . The consultation sought to address five issues that have arisen since the Orphan Regulation came into effect in January 2000: Clarifying the meaning of "significant benefit" How to apply the Orphan Regulation to emerging diseases, such as Ebola, that are not present within...
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    EMA Offers New Guidance on Type II Variations and MA Extensions of Orphan Drugs

    The European Medicines Agency (EMA) has updated its post-authorization guidance on how extensions of marketing authorizations and type II variations may impact orphan-designated medicines. The updates follow the publication of a new  notice by the European Commission on the application of Articles 3, 5 and 7 of Regulation (EC) No. 141/2000 on orphan medicinal products . In this notice, the European Commission provides guidance on the application of the Orphan Regulation,...
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    FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments

    The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for 21 new clinical trials over the next four years to boost the development of rare disease treatments. The grants, awarded to researchers from academia and industry at domestic and international clinical sites, are part of FDA’s Orphan Products Clinical Trials Grants Program, created in 1983, which has provided more than $370 million (including $19 million ...
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    FDA Finalizes Guidance on Tropical Disease PRVs, Offering a Number of Clarifications

    The US Food and Drug Administration (FDA) on Wednesday finalized guidance on tropical disease priority review vouchers (PRVs), clarifying when a voucher can be used, whether drugmakers are “guaranteed” a six-month review when using a voucher and whether FDA can remove a tropical disease from the list of considered diseases (it can’t). This finalization of the 2008 draft guidance includes 25 questions and answers and includes the following substantive changes based on pu...
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    Priority Review Voucher Fees to Decline in FY 2017

    The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year. Tropical Disease Priority Review Voucher User Fee Year Voucher Fee FY 2011 $4,582,000 FY 2012 $5,280,000 FY 2013 $3...
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    Industry Weighs in on FDA Draft Guidance on Infectious Disease NGS-Based Diagnostics

    Diagnostic companies Illumina, Roche, AstraZeneca and others are offering a peek inside how they view next-generation sequencing (NGS) diagnostic devices, with the release of 20 comments on recent US Food and Drug Administration (FDA) draft guidance. "Next generation sequencing has the capability to replace previous methods with a single approach to accomplish what might have required several different tests in the past," FDA explains. "In contrast to human sequencing di...