EU/3/15/1569 - orphan designation for treatment of idiopathic hypersomnia
pentetrazol
OrphanHuman
Overview
On 11 November 2015, orphan designation (EU/3/15/1569) was granted by the European Commission to Dr Jens Steinbrink, Germany, for pentetrazol for the treatment of idiopathic hypersomnia.
The sponsorship was transferred to Balance Therapeutics, Limited, United Kingdom, in January 2016.
The sponsorship was transferred to Pharma Gateway AB, Sweden in October 2019.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2020 on request of the Sponsor.
Idiopathic hypersomnia is a sleep disorder characterised by excessive daytime sleepiness. Idiopathic means that the cause of the disease is unknown.
People with this condition struggle to stay awake during the day and feel the need to take frequent long naps. These may be prolonged or at inappropriate times, such as during a meal or a conversation, and generally don't provide any relief from the sleepiness. Most people with idiopathic hypersomnia also sleep for more than 10 hours a night and struggle to wake in the morning, because they feel very drowsy and confused upon waking.
Idiopathic hypersomnia is a long-term debilitating disease because the need for sleep during the day interferes with normal life.
At the time of designation, idiopathic hypersomnia affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 154,000 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).
*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 512,900,000 (Eurostat 2015).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of idiopathic hypersomnia. Patients with idiopathic hypersomnia usually received advice on lifestyle changes to help regulate their sleeping pattern.
The medicine pentetrazol has been used for several years in the treatment of conditions such as respiratory disorders and low blood pressure.
Pentetrazol is a 'GABA-receptor antagonist': it blocks the action of a substance in the brain called GABA, which is thought to play a role in promoting sleep and whose function is believed to be elevated in idiopathic hypersomnia. By blocking GABA's function, the medicine is expected to reduce excessive daytime sleepiness.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with idiopathic hypersomnia were planned.
At the time of submission, the medicine was not authorised anywhere in the EU for idiopathic hypersomnia. Orphan designation of the medicine has been granted in the United States for idiopathic hypersomnia.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 October 2015 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
- pentetrazol
- Intended use
- Treatment of idiopathic hypersomnia
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/15/1569
- Date of designation
- Sponsor
Pharma Gateway AB
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: