EMA opinion gives hope

EMA opinion gives hope for treatment of Prader-Willi syndrome

In January 2012, the Committee for Orphan Medicinal Products (COMP) recommended the granting of an orphan designation for carbetocin, a first in the orphan designation opinion, for the treatment of Prader-Willi syndrome (PWS).

This seriously debilitating disease currently has no approved treatment.
Patients with PWS often have a significantly reduced life span and require lifelong care.

Prader-Willi syndrome causes a wide range of symptoms, such as:

• a constant desire to eat food resulting in permanent hunger and leading to dangerous obesity,
• impaired function of the gonads,
• learning difficulties and behavioural problems.

Orphan designation gives the sponsor of carbetocin access to incentives that encourage and support the development of this medicine.

The assessment of the designation application for carbetocin was supported by the expert advice from patient representatives from (EURORDIS), who confirmed the high unmet medical need in the management of patients with Prader-Willi syndrome.

This breakthrough also offers treatment possibilities for other food associated rare diseases, autistism, and some psychotic behaviour problems.

• Read the Announcement on EMA website
• More about Prader-Willi syndrome
• Read more about Orphan Drug Development and the role of EURORDIS