62 guidances, approvals and safety alerts affecting rare_diseases, collected from official agency sources. Showing the 30 most recent.
Committee for Advanced Therapies (CAT) is scheduled to convene 7–9 October 2026 at the European Medicines Agency in Amsterdam to support the EU’s assessment…
CVMP is scheduled to meet on 6–8 October 2026 to prepare the European Medicines Agency’s opinions on veterinary medicines-related questions.
COMP scheduled meeting (6–8 October 2026) will review applications for orphan medicinal product designation for medicines intended for diagnosis, prevention…
Establishes an Implementation Advisory Group (IAG) to support execution of Canada’s National Strategy for Drugs for Rare Diseases, providing a structured forum…
Launches WHO’s prequalification procedure for medical devices, signposting a new pathway for manufacturers to seek WHO endorsement and broader market access.
Launches WHO PQT eCTD submission capability via the ePQS Portal, enabling a harmonized electronic format to improve dossier lifecycle management and streamline…
Expands the WHO API prequalification Expression of Interest (EOI) process to include additional APIs—Hydrocortisone (sodium succinate), Dactinomycin…
Clarifies WHO prequalification expectations for plain-language descriptions of the visual appearance of oral dosage forms in WHOPARs, improving consistency and…
Launches the first WHO PQT invitation for manufacturers to submit an Expression of Interest (EOI) for evaluation of hydroxyurea products for the treatment of…
Adds hydroxyurea API for products used to treat sickle cell disease to the WHO Prequalification Unit (PQT) expression of interest (EOI) pathway for API…
Promotes structured collaboration to address gaps in real-world data (RWD) collection for Duchenne muscular dystrophy, with the goal of generating meaningful…
Convenes the EMA’s Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) to coordinate oversight actions related to medicines shortages…
Provides a CHMP reflection framework for sponsors planning clinical development of medicinal products intended for systemic sclerosis, with particular emphasis…
Facilitates a structured dialogue between EMA and Affordable Medicines Europe to exchange views on topics of common interest within EMA’s annual bilateral…
Schedules the COMP meeting (8–10 September 2026) at the European Medicines Agency in Amsterdam to support review of applications for orphan medicinal product…
Extends the use of a prior intimation mechanism for specified bioavailability (BA) and bioequivalence (BE) studies submitted to CDSCO as part of approval of…
Simplifies Swissmedic application procedures for changing a veterinary medicinal product’s status between its main authorisation and an export licence.
Introduces a draft amendment notice for Korea’s MFDS regulations on the safety of pharmaceuticals and related products.
Clarifies FDA expectations for demonstrating therapeutic equivalence for approved drug products, with emphasis on the evidentiary basis used to support…
Clarifies FDA expectations for potency assessment strategies for active immunotherapy products, which may influence how sponsors design potency assays and…
Publishes new WHO Public Inspection Reports (WHOPIRs), expanding transparency on WHO-inspection outcomes for regulated product manufacturers.
Updates the WHO Emergency Use Listing (EUL) process status for Bundibugyo virus disease (BDBV) in vitro diagnostics, indicating active intake and assessment of…
Amends India’s Drugs and Cosmetics Rules, 2017 to incorporate EU countries within the scope of Rule 63 and extends/aligns Quality Management System (QMS)…
Common Submission Format guidance for importing and registering drugs (bulk and finished formulations) in India is intended to standardize how dossiers are…
Establishes a forthcoming EMA CVMP guideline concept that will tighten quality and safety expectations for RNA interference (RNAi) and RNA antisense…
Prohibits the marketing/approval of 16 fixed-dose combinations (FDCs) under specified Gazette notifications, potentially affecting existing and pending…
Human Tissue Safety Regulations amendment (draft) is being published for public comment, indicating potential updates to Korea’s regulatory requirements…
COMP meeting is scheduled for 14–16 July 2026 and will review orphan medicinal product designation applications for medicines intended for diagnosis…
Initiates an MFDS legislative notice proposing amendments to the Enforcement Rules under the Act on the Management of Sanitary Products.
Fostering regulatory support across the medicines lifecycle, this EMA stakeholder platform meeting will shape how scientific advice, qualification of novel…
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