• Feature ArticlesFeature Articles

    Shaking Palsy, aka Parkinson's Disease, is Still a Mystery

    This article discusses disease etiology, history and symptoms of Parkinson's disease as well as ongoing research and new treatment options. Introduction There are hundreds of diseases named after a person. These are called "eponymous diseases," and a small sample includes Crohn's disease, Salmonellosis, Huntington's disease, Hodgkin's lymphoma, Lou Gehrig's disease, Tourette's syndrome and the well-known Alzheimer's disease and Parkinson's Disease (PD). PD is one of...
  • Regulatory NewsRegulatory News

    FDA Issues Disease-Specific Draft Guidance for Pediatric GERD

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance detailing clinical considerations for drugmakers looking to develop products to treat pediatric gastroesophageal reflux disease (GERD). FDA says the draft is meant to be a starting point for discussions between the agency, drugmakers, academia and the public and sets out the agency's current thinking on potential study populations, endpoints and clinical pharmacology issues related to dosing. Sp...
  • Regulatory NewsRegulatory News

    Guest Editorial: Does Nutrition Have a Role in Disease Management?

    This editorial is meant to encourage discussion and thinking about the value of diet and nutrition in the management of disease and a regulatory framework that would be useful in the US to protect public health and provide benefit to individuals who live with chronic disease or an increased risk of such diseases. Introduction The current regulatory approach, with respect to food and nutrition, tends to categorize individuals as consumers or patients and yet these stat...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    EMA and FDA Look to Facilitate Development of Gaucher Disease Treatments

    The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Monday released a new strategy document to aid in the development of new treatments for Gaucher disease, a rare lysosomal storage disorder. Given the limited number of patients worldwide with Gaucher disease, EMA and FDA put forth two possible approaches to better facilitate investigations of such products: Extrapolation across age groups within a single drug development program...
  • Regulatory NewsRegulatory News

    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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    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. ...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
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    EMA and FDA Set Up New Working Group on Rare Diseases

    The new collaboration between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) announced Monday will improve the sharing of information on various aspects of the development and scientific evaluation of medicines for rare diseases. According to EMA, rare diseases affect 30 million people in the EU and approximately the same number in the US, though each disease individually concerns a limited number of patients. And since only a limited n...