Orphan designation: aganirsen Prevention of corneal graft rejection, 20/04/2026 Positive

Overview

This medicine was designated as an orphan medicine for the prevention of corneal graft rejection in the European Union on 20 April 2026.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

• EU Clinical Trials Register
• ClinicalTrials.gov

Key facts

Active substance

aganirsen

Intended use

Prevention of corneal graft rejection

Orphan designation status

Positive

EU designation number

EU/3/26/3225

Date of designation

20/04/2026

Sponsor

Laboratoires Kol

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

• List of opinions on orphan medicinal product designation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

• European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
• Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

• European Commission: Community Register of orphan medicinal products