EU/3/20/2313 - orphan designation for diagnosis of progressive supranuclear palsy
2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine
OrphanHuman
Overview
On 21 August 2020, orphan designation EU/3/20/2313 was granted by the European Commission to Life Molecular Imaging GmbH, Germany, for 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine for the diagnosis of 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, personality changes and dementia (loss of intellectual function). The disease usually starts in people aged over 60 years and gradually gets worse over a number of years.
Patients with PSP have abnormal tangles of a protein called 'tau' in their brain, which are thought to cause the gradual deterioration of brain tissue seen in these patients.
PSP is a debilitating and life-threatening disease that leads to parkinsonism, paralysis and premature death.
At the time of designation, the number of patients eligible for diagnosis of progressive supranuclear palsy was estimated to be approximately 1.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 93,000 people*, and is below the ceiling 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).
*For the purpose of the designation, the number of patients eligible for diagnosis is estimated and assessed on the basis of data from the European Union, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
No satisfactory methods of diagnosis of PSP were authorised at the time of application. Magnetic resonance imaging (MRI) was used to support diagnosis based on clinical signs and symptoms in late stages of the disease.
The medicine is a radiopharmaceutical (a medicine that emits a small amount of radioactivity) that binds specifically to tau protein in the patient’s brain. The resulting build-up of radiation makes it possible to see the protein tangles using a technique called positron emission tomography (PET) scan and help to determine which patients are suffering from the condition.
At the time of submission of the application for orphan designation, clinical trials with 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine in patients with progressive supranuclear palsy were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the diagnosis of PSP or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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
- 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine
- Intended use
- Diagnosis of progressive supranuclear palsy
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2313
- Date of designation
- Sponsor
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:
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: