Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
Be among the first to receive Kisho Intelligence Briefings
Showing 501–520 of 2553 stories
The CDC announces informational webinars for potential host sites of the Public Health Associate Program (PHAP). These sessions will guide applicants through the application process and provide essential details.
FDA issues a priority review voucher for KYGEVVI (doxecitine and doxribtimine), a product for a rare pediatric disease. This voucher can expedite the review process for future drug applications, enhancing the sponsor's ability to bring new therapies to market.
FDA has approved RHAPSIDO (Remibrutinib) under a rare pediatric disease priority review voucher, highlighting the agency's commitment to expedite treatments for rare conditions. This approval underscores the importance of priority review vouchers in bringing innovative therapies to market.
The FDA has released draft guidance recommending the use of minimal residual disease (MRD) and complete response (CR) as primary endpoints in clinical trials for multiple myeloma. This guidance aims to facilitate accelerated approval processes for new therapies targeting this disease.
FDA has released a draft guidance titled 'M4Q(R2) The Common Technical Document for the Registration of Pharmaceuticals for Human Use: Quality' aimed at creating a globally harmonized framework for pharmaceutical registration. This guidance is part of the International Council for Harmonisation's efforts to streamline regulatory processes.
The FDA has issued Emergency Use Authorizations (EUA) for certain medical devices related to COVID-19, as mandated by the Federal Food, Drug, and Cosmetic Act. This announcement includes the requirement for the FDA to publish notices of each authorization in the Federal Register.
The Health Information Technology Advisory Committee (HITAC) will hold public meetings throughout 2026 to identify priorities for standards adoption and provide recommendations to the Assistant Secretary for Technology Policy. This initiative is part of the ongoing implementation of the 21st Century Cures Act.
A recent systematic review confirms that the use of paracetamol during pregnancy does not increase the risk of autism, ADHD, or intellectual disability. The EMA maintains that paracetamol is a crucial option for managing pain or fever in pregnant women based on extensive scientific data.
NHS Scotland has approved Exagamglogene autotemcel, a CRISPR gene therapy for severe sickle cell disease, promising durable reduction in vaso-occlusive crises. This approval is contingent on a Patient Access Scheme, allowing for confidential pricing arrangements for this high-cost therapy.
The HHS is undergoing a review under the Paperwork Reduction Act, which may impact data collection processes. This review could affect how health-related information is gathered and reported.
The HHS is currently undergoing a review under the Paperwork Reduction Act, which may impact data collection processes. Stakeholders should monitor developments as changes could affect reporting requirements.
The Administration for Children and Families seeks a 3-year extension for the ACF-204 report on Temporary Assistance for Needy Families and State Maintenance-of-Effort programs, with no substantive changes but updated instructions. This report is crucial for tracking state compliance and funding allocation.
The Federal Register announces that an agency is undergoing a review under the Paperwork Reduction Act. This process may impact regulatory requirements but lacks immediate implications for stakeholders.
The National Library of Medicine has announced partially closed meetings, which may impact future research funding and priorities. Stakeholders should monitor these developments for potential implications on rare disease initiatives.
AgomAb Therapeutics and SpyGlass Pharma have launched their IPOs as part of the 2026 class, joining other biopharma companies like Veradermics and Eikon Therapeutics. AgomAb's lead asset, AGMB-129, targets fibrostenosing Crohn’s disease and is an orally available small-molecule blocker of the ALK5 protein.
EMA outlines key recommendations for the authorization and safety monitoring of veterinary medicines by 2025. This overview includes 30 specific recommendations aimed at enhancing regulatory frameworks.
Global Genes and the University of Notre Dame have launched the Research Acceleration Program to enhance collaboration in rare disease research. This initiative aims to foster partnerships that can lead to innovative solutions for rare diseases.
The Committee for Veterinary Medicinal Products (CVMP) issued a positive opinion for Elanco's Lotilaner / Milbemycin, advancing its marketing authorization. This decision reflects ongoing efforts to enhance veterinary medicinal options.
The EMA's Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting from January 12-15, 2026, focusing on risk management of medicines. While specific decisions were not highlighted, the meeting underscores ongoing regulatory oversight.
The Light Up for Rare initiative aims to enhance awareness and advocacy for rare diseases by illuminating landmarks globally on Rare Disease Day. The organization envisions future light-ups to focus on themes like patient safety and involvement, encouraging organizations to participate by lighting their own buildings.
Get the week's most important rare disease developments — pipeline moves, policy changes, and funding signals — delivered to your inbox every Monday.
Free weekly briefing. Unsubscribe anytime.