EMA recommends granting marketing authorization for Teizeild (teplizumab) to delay the onset of stage 3 type 1 diabetes in adults. This first-in-class treatment represents a significant advancement in diabetes management.

The U.S. Food and Drug Administration today announced a pilot program designed to streamline communications with sponsors following formal meetings.

EMA's Emergency Task Force is enhancing its scientific advice process for medicines targeting public health threats, including antimicrobial resistance. This new approach aims to provide clearer guidance on clinical trial design, facilitating faster development and approval of critical therapies during health emergencies.

The U.S. Food and Drug Administration today announced it is taking action to approve new labeling submitted by the company that includes the addition of a Boxed Warning, the agency’s most prominent safety warning, to Elevidys (delandistrogene moxeparvovec-rokl), and that the indication section of th

NORD has launched a new Request for Proposals (RFP) to implement two patient registries on the IAMRARE® platform, funded by RDCA-DAP®. This initiative aims to enhance data collection and support for rare disease communities.

Over 110 individuals affected by rare diseases gathered in Milwaukee for the 'Living Rare, Living Stronger' event, fostering connections among patients, caregivers, and advocates. This initiative highlights the importance of community support in navigating the challenges of rare diseases.

The EMA's CHMP recommended ten new medicines for approval during its November 2025 meeting, indicating a positive trend in drug development. Specific details on the drugs and their indications were not disclosed in the highlights.

The U.S. government has reopened following the signing of a continuing resolution by the President on November 12. This agreement funds most government operations, ensuring stability for ongoing health policies and programs.

The U.S. Department of Health and Human Services (HHS) and the U.S. Food and Drug Administration (FDA) today announced that Richard Pazdur, M.D., has been appointed director of the Center for Drug Evaluation and Research (CDER). Pazdur is a 26-year veteran of the FDA.

The U.S. Department of Health and Human Services (HHS) today announced historic action to restore gold-standard science to women’s health.

The EU's upcoming 2028-34 budget will significantly impact funding for rare diseases, with decisions in the coming months crucial for resource allocation. Stakeholders are urged to advocate for improved financial frameworks to support those living with rare conditions.

The U.S. Food and Drug Administration today announced six additional awardees under the Commissioner’s National Priority Voucher (CNPV) pilot program.

The U.S. Food and Drug Administration issued 18 warning letters to owners of websites illegally marketing unapproved and misbranded botulinum toxin products, commonly called Botox.

11 people from 7 states have gotten sick from the same strain of Salmonella.

CDC urges people to check their refrigerators and freezers for recalled foods. Do not eat them.

EURORDIS celebrates the 40th workshop of its Round Table of Companies, focusing on the integration of patient experience data into medicines regulation. This initiative aims to enhance the role of patient insights in the drug development process.

Global Genes hosted the RARE Advocacy Exchange Session 8 on October 16, 2025, focusing on educating patients about their rights in the rare disease landscape. This session aims to empower individuals and organizations advocating for rare disease awareness and support.

The EveryLife Foundation has submitted comments to the FDA on the Reauthorization of Medical Device User Fee Agreements (MDUFA) for fiscal years 2028 and beyond. This initiative could impact the regulatory landscape for medical devices affecting rare diseases.

Over 190 organizations, led by the EveryLife Foundation, support the Give Kids a Chance Act of 2025, which aims to reauthorize the Rare Pediatric Disease Priority Review Voucher program for five years. This legislation is crucial for accelerating the development of treatments for rare pediatric diseases.

The EU Biotech Act is poised to significantly impact rare disease progress, with EURORDIS outlining key priorities for its implementation. Stakeholders are urged to ensure that the Act translates into tangible benefits for the rare disease community.