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Orphan designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in April 2013 on request of the sponsor.

On 23 March 2010, orphan designation (EU/3/10/728) was granted by the European Commission to FGK Representative Service GmbH, Germany, for davunetide for the treatment of progressive supranuclear palsy.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is progressive supranuclear palsy?

Progressive supranuclear palsy (PSP), which is also known as Steele-Richardson-Olszewski syndrome, is a rare disease that involves the gradual deterioration of parts of the brain. Symptoms include loss of balance with unexplained falls, stiffness, difficulty moving the eyes (particularly up and down), difficulty swallowing, personality changes and dementia (loss of intellectual function). The disease usually starts in people aged over 40 years and gradually gets worse over a number of years.

PSP is a debilitating and life-threatening disease that leads to a progressive inability to move and poor long-term survival.

What is the estimated number of patients affected by the condition?

At the time of designation, PSP affected approximately 1 in 10,000 people in the European Union (EU). This is equivalent to a total of around 51,000 people*, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of PSP. Because of their tendency to fall, patients were often offered walking aids, as well as special glasses to help them to look down. Physiotherapy was used to keep the joints flexible. For patients unable to swallow, a feeding tube leading through the tummy to the stomach was used. Medicines developed to treat Parkinson’s disease were also used in some PSP patients, but their effect was usually limited, and did not last long.

How is this medicine expected to work?

PSP is thought to be caused by proteins called tau sticking together and forming tangled filaments. These filaments build up in brain cells, damaging various areas of the brain. The way that davunetide works in PSP is not fully understood, but it is thought to prevent tau sticking together, reducing the damage to brain cells.

What is the stage of development of this medicine?

The effects of davunetide have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with davunetide had been started in patients with PSP.

At the time of submission, davunetide was not authorised anywhere in the EU for PSP or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 January 2010 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria
  • the seriousness of the condition;
  • the existence of alternative methods of diagnosis, prevention or treatment;
  • either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Davunetide</p>
Active substanceDavunetide
Medicine Name
Disease/conditionTreatment of progressive supranuclear palsy
Date of decision23/03/2010
Orphan decision numberEU/3/10/728

Review of designation

Sponsor’s contact details

FGK Representative Service GmbH
Heimeranstrasse 35
80339 München
Tel. + 49 89 893 119 22
Fax + 49 89 893 119 20

Patients’ organisations

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

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