ACIP today voted to recommend individual-based decision-making for hepatitis B vaccine.

The Committee for Veterinary Medicinal Products (CVMP) issued a positive opinion for Elanco GmbH's Varenzin (molidustat), indicating progress towards marketing authorization. This highlights ongoing developments in veterinary medicinal products.

The U.S. Food and Drug Administration today approved a new indication for Breyanzi (Lisocabtagene maraleucel) as the first Chimeric Antigen Receptor (CAR) T-cell therapy in the U.S.

NORD calls on Congress to swiftly reauthorize the Rare Pediatric Disease Voucher Program, which has facilitated 63 FDA vouchers for 47 rare pediatric diseases, including 43 with no prior treatments. This program is crucial for advancing therapies for underserved children.

Today, the U.S. Food and Drug Administration conditionally approved Exzolt Cattle-CA1 (fluralaner) topical solution for the prevention and treatment of New World screwworm (NWS) larval infestations, and the treatment and control of cattle fever tick in beef cattle 2 months of age and older and repl

The U.S. Food and Drug Administration (FDA) today announced that Tracy Beth Høeg, M.D., Ph.D., has been appointed acting director of the Center for Drug Evaluation and Research (CDER).

The U.S. Food and Drug Administration today granted approval for Avance (acellular nerve allograft-arwx) in surgical implantation.

The National Organization for Rare Disorders (NORD) has expanded its network by adding seven new Rare Disease Centers of Excellence, bringing the total to 46 institutions. This initiative aims to enhance rare disease care and foster research collaboration across the country.

The U.S. Food and Drug Administration, in coordination with the U.S. Department of Justice, announced today that the U.S. Marshals Service seized approximately 73,000 units of 7-hydroxymitragynine (7-OH) products—valued at roughly $1 million—from three firms in Missouri.

Today, the U.S. Food and Drug Administration (FDA) issued draft guidance outlining specific product types for which the FDA believes six-month non-human primate toxicity testing can be eliminated or reduced.

The House of Representatives has passed the Give Kids a Chance Act (H.R. 1262), which aims to enhance policies that support rare disease research and treatment. The EveryLife Foundation for Rare Diseases commends this legislative progress as a significant step towards improving outcomes for affected children.

The U.S. Food and Drug Administration today announced the deployment of agentic AI capabilities for all agency employees.

The TSC Alliance and Dup15q Alliance emphasize the critical role of awareness in improving early diagnosis and outcomes for infantile spasms during Awareness Week. Their collaboration aims to educate families on symptoms and the importance of timely intervention.

AFM-Téléthon 2025 is set to raise awareness and funds for rare diseases, continuing its legacy of supporting research and patient advocacy. The event aims to engage the community and highlight the importance of rare disease initiatives.

EURORDIS announces the ERN ReCONNET event focused on sharing good clinical practices for rare diseases in 2025. This initiative aims to enhance collaboration among stakeholders in the rare disease community.

EURORDIS hosts a webinar focusing on the UnDiagnosed Patients Working Group under ERN LUNG, aiming to enhance collaboration and support for patients with undiagnosed lung diseases. This initiative highlights the importance of patient engagement in rare disease research and advocacy.

The European Commission has extended the deadline for applications to participate in EMA’s Paediatric Committee, now due by December 5, 2025. This initiative aims to include civil society representatives from patient and healthcare professional organizations.