EU/3/22/2725: Orphan designation for the diagnosis of ATTR amyloidosis

Transthyretin (TTR) amyloidosis is a rare and life-threatening disease in which misshaped TTR proteins build up in organs, including the heart, to form fibrous clumps called amyloid deposits, leading to organ dysfunction.

Iodine (124I) evuzamitide is a radioactive substance for use with an imaging method called positron emission tomography (PET). It consists of a radioactive element, iodine (124I), attached to a protein that binds to amyloid deposits. After it binds to the deposits, the radiation it emits can be detected by a PET scanner, enabling doctors to see the location and extent of amyloid deposits in the patient’s body.

Based on description provided by sponsor

At the time of submission of the application for orphan designation:

• The evaluation of the effects of the medicine in experimental models was ongoing.
• Clinical trials with the medicine in patients with ATTR amyloidosis were ongoing.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

• scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;

• market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

• there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

• Orphan designation: Overview

• Rare diseases, orphan medicines: Getting the facts straight