• Regulatory NewsRegulatory News

    Euro Convergence: How orphan drug suppliers can win labelling exemptions

    A strong local presence in EU markets is needed when seeking labelling exemptions that help reduce the cost of providing orphan products, according to experts who spoke at RAPS Euro Convergence 2021.   Manufacturers of orphan drugs can gain exemptions from some labelling requirements at an EU level and from individual member states, a potentially valuable flexibility for companies that make orphan drugs targeting very small numbers of patients. Such products face speci...
  • Regulatory NewsRegulatory News

    CHMP recommends eight new medicines in April, including two orphans

    In its April meeting, the European Medicines Agency’s (EMA’s) human medicines committee recommended eight new medicines for approval, including three new non-orphan medicinal products and two orphan medicines.   EMA’s Committee for Medical Products for Human Use (CHMP) also issued positive opinions for extensions of therapeutic indications for nine medicines, bringing the total new indications for 2021 to 23.   Leo Pharma’s Adtralza (tralkinumab) was recommended ...
  • Regulatory NewsRegulatory News

    Orphan product designation requests climb in the US, remain steady in the EU

    The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.   There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019...
  • Regulatory NewsRegulatory News

    FDA on Rare Disease Day: New initiatives and an upcoming conference

    On Monday, the US Food and Drug Administration (FDA) announced a series of efforts and a day-long conference centered on addressing the unmet needs of patients with rare diseases. FDA leaders announced the initiatives in recognition of Rare Disease Day, observed on February 28.   A new request for applications (RFA) issued by FDA augments the agency’s Orphan Products Grants Program. The RFA is seeking natural history studies to address unmet needs in rare diseases, l...
  • Regulatory NewsRegulatory News

    FDA launches online orphan submissions portal

    The US Food and Drug Administration (FDA) on Monday launched a new cloud-based submission portal for orphan drug designation requests, shifting away from the current paper and CD-based submission process. The new portal, first announced in January, is part of the agency’s orphan drug technology modernization effort and fits in with its broader technology modernization plans. The shift to electronic submissions follows a decade of increasing orphan drug designation reques...
  • 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...
  • Regulatory NewsRegulatory News

    Stakeholders call for regulatory clarity in rare disease research network

    Stakeholders weighing in on a proposed rare disease clinical trials network called for regulatory clarity, smart use of existing resources, and a move toward harmonized trial standards and assessments.   As part of the launch of the US Food and Drug Administration (FDA)’s Rare Disease Cures Accelerator , the agency asked for stakeholder input on how FDA and other agencies can achieve a more cooperative approach in supporting the drug development pipeline for rare dise...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    FDA officials update on orphan drugs, gene therapies at DIA

    Officials from the US Food and Drug Administration (FDA) discussed the agency’s recent efforts to support the development of products to treat rare diseases during a session at DIA’s Global Annual Meeting on Wednesday.   Orphan and rare pediatric disease designations   While the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.   Janet Maynard, director of F...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    FDA seeks input on rare disease clinical trials network

    To further the establishment of a rare disease clinical trials network, the US Food and Drug Administration (FDA) is asking for input from the public and a broad array of stakeholders.   In a press release, the agency noted that it is “looking to provide a more cooperative approach” in supporting the drug development pipeline for rare diseases.   The call for information comes as the FDA is launching a Rare Disease Cures Accelerator . This accelerator will establi...