EU/3/17/1883 - orphan designation for treatment of Creutzfeldt-Jakob disease
Pentamer formyl thiophene acetic acid
OrphanHuman
Overview
On 20 June 2017, orphan designation (EU/3/17/1883) was granted by the European Commission to NeuroScios GmbH, Austria, for pentamer formyl thiophene acetic acid (also known as NSC500) for the treatment of Creutzfeldt-Jakob disease.
Creutzfeldt-Jakob disease is a disease of the brain which causes worsening dementia, difficulty with movement and problems with vision. It is caused by the incorrect folding of a protein known as a prion. The incorrect folding ('misfolding') makes the prion proteins clump together and build up in brain cells, damaging the cells.
Early symptoms of the disease include failing memory, behaviour changes, inability to control muscles (lack of coordination) and changes in sight. As the disease worsens, the patient may develop uncontrolled movements, blindness, weakness in the arms and legs and fall into a coma. Patients with the disease usually die within a year.
Creutzfeldt-Jakob disease is a fatal disease which is seriously debilitating because of the rapid progression of brain damage with loss of mental function, problems of coordination, personality changes and failing sight.
At the time of designation, Creutzfeldt-Jakob disease affected less than 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 5,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 515,700,000 (Eurostat 2017).
No satisfactory methods were authorised for the treatment of Creutzfeldt-Jakob disease at the time of application. Treatment was aimed at reducing pain and other symptoms and making the patient as comfortable as possible.
This medicine attaches to the misfolded prion protein in brain cells and interferes with the way these proteins clump together. It is expected that, by interfering with this clumping, the medicine makes the abnormal prion proteins more soluble and less damaging to brain cells. This is expected to slow down the progression of Creutzfeldt-Jakob disease and so delay the symptoms from worsening.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission, no clinical trials with the medicine in patients with Creutzfeldt-Jakob disease had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for Creutzfeldt-Jakob disease 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 12 May 2017 recommending the granting of this designation.
- 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
- Active substance
- Pentamer formyl thiophene acetic acid
- Intended use
- Treatment of Creutzfeldt-Jakob disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1883
- Date of designation
- Sponsor
NeuroScios GmbH
Willersdorfer Straße 7
8061 St. Radegund/Graz
Austria
Tel. +43 3132 404 44 11
E-mail: office@NeuroScios.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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:
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.
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: