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  • Regulatory NewsRegulatory News

    FDA orphan drug updates at NORD Summit

    Officials from the US Food and Drug Administration (FDA) provided updates on the agency’s efforts to support the development of medical products for rare diseases at the National Organization for Rare Disorders (NORD) Summit this week.   In his keynote address on Thursday, FDA Commissioner Stephen Hahn said that FDA had already approved 51 orphan indications through July of this year and acknowledged that the agency’s Office of Orphan Products Development (OOPD) has se...
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    European Commission reviews impact of orphan, pediatric regulations

    The European Commission on Tuesday published the results of a yearslong evaluation of the EU’s orphan and pediatric medicines regulations it says will be used to guide future legislative changes and shape the EU pharmaceutical strategy.   The more than 100-page evaluation reviews the positive impacts and shortcomings of the orphan regulation, Regulation (EC) No 141/2000 , and the pediatric regulation, Regulation (EC) No 1901/2006 , based on external studies and var...
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    Experts address clinical challenges for ultra-rare diseases at BIO

    A panel of experts at BIO Digital on Wednesday discussed some of the challenges and regulatory considerations for sponsors developing treatments for ultra-rare diseases where clinical trials could involve a very small number of patients.   When asked where companies should begin their development for extremely rare diseases, Brad Glasscock, global vice president and head of global regulatory affairs at BioMarin said, “First and foremost I think is understanding whether...
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    Orphan drug incentives reviewed at BIO

    Orphan drug designation has been a fruitful pathway for drug development, but unmet need remains. Whether changes are needed to the legislation, and how orphan drug research can incorporate patient-focused drug development principles and make room for modern-day collaboration were topics for expert discussion at an orphan drug-focused session during BIO Digital 2020.   The future looks bright for drug development for rare diseases, said session moderator Natasha Bonhom...
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    FDA to Congress: Pediatric Information Lacking in 36% of Relevant Orphan Drug Labels

    In a report to Congress, the US Food and Drug Administration said that there is a public health need for additional pediatric information in labeling for over one-third of approved orphan indications that are relevant in the pediatric population. FDA research, conducted as part of the FDA Reauthorization Act of 2017  (FDARA), found that of all drugs that were approved for an orphan indication between 1 April 1999 and 31 August 2018, a total of 548 orphan indications we...
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    Drugmaker Petitions FDA to Revoke Sublocade Orphan Designation

    A citizen petition filed last week by law firm Goodwin Procter on behalf of Pennsylvania-based drugmaker Braeburn calls on the US Food and Drug Administration (FDA) to revoke the orphan designation granted to Indivior’s opioid use disorder treatment Sublocade (buprenorphine extended-release injection).   “Sublocade is not now, nor was it ever a bona fide orphan drug, particularly since more than two million Americans currently are afflicted by opioid addiction,” the pe...
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    Patient Groups, Industry Seek Changes to Rare Disease Drug Guidance

    Patient groups, drugmakers and other organizations are calling for changes to the US Food and Drug Administration’s (FDA) recently revised draft guidance on developing drugs to treat rare diseases.   The 24-page draft guidance was revised last February after first being published in 2015. The revision included updates to the agency’s approach to natural history studies, a discussion of issues for evaluating biomarkers for consideration as surrogate endpoints and a ne...
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    European Commission Consults on Orphan Drug Applications

    The European Commission on Tuesday launched a targeted consultation to provide input on a revision of its guideline on the content and format of applications for orphan drug designation, as well as the transfer of designations between sponsors.   "The comments received will be taken into account by the European Commission in the finalization of the guideline on the format and content of application for designation as orphan medicinal products and on the transfer of des...
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    GAO Raises Questions on FDA’s Orphan Drug Designation Process

    The Government Accountability Office (GAO) announced Friday that it has found that the US Food and Drug Administration (FDA) does not always ensure that all information is consistently recorded and evaluated when making orphan drug designation determinations. The missing of these critical steps by the agency in some cases means that FDA reviewers do not fully understand the context of a drug’s intended use for the rare disease, GAO said. “The Commissioner of FDA shou...
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    EMA Begins Publishing Reports on Whether Approved Medicines Still Qualify as Orphans

    The European Medicines Agency (EMA) on Wednesday announced it has begun publishing reports detailing its decision-making on whether newly approved drugs to treat rare diseases still qualify for orphan designation when they receive marketing authorization. In the EU, drugs intended to treat diseases affecting fewer than five in 10,000 people in the EU qualify for orphan designation, which provides authorized medicines with ten years marketing exclusivity and reduced fees....
  • European Regulatory Roundup: France Targets 80% Biosimilar Penetration by 2022 (4 January 2018)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news . Ireland Offers to Waive Certain Fees to Ease Impact of Brexit Ireland is offering to waive the fee for certain Brexit-triggered regulatory requests. The offer will allow companies to change their reference member state (RMS) from the United Kingdom to Ireland for free. Officials from the Health Products Regulatory Authority (HPRA) of Ireland made the offer in guides on ...
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    FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements

    The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions. Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of th...