EMA Publishes Guidance on Applying for Expansions of Orphan Indications and Drugs

The European Medicines Agency published new guidance on whether less comprehensive data can be used to support a new indication for a previously approved orphan drug product.

The EMA also detailed whether sponsors need to confirm an orphan designation when applying for label variations that may significantly impact the product’s quality, safety or efficacy, in a revision of its post-authorization guidance.

If an orphan product application concerns a new therapeutic indication or a modification of an existing one, in order to ensure that a marketing authorization only covers indications that fulfill the orphan designation criteria, a review may be required by the Committee for Orphan Medicinal Products, the EMA said.