EU/3/21/2553 - orphan designation for treatment of spinocerebellar ataxia
Troriluzole hydrochloride
OrphanHuman
Overview
This medicine was designated as an orphan medicine for the treatment of spinocerebellar ataxia in the European Union on 10 December 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Troriluzole is a drug that normalizes levels of glutamate, a neurochemical that activates cells. Troriluzole increases glutamate clearance from nerve synapses, the spaces between neurons where messages are transmitted. Troriluzole may also affect certain potassium channels on the surface of nerve cells. In spinocerebellar ataxia, nerve cells, known as Purkinje neurons, in a part of the brain called the cerebellum, die. Over activation of these neurons via excessive glutamate and dysregulation of certain potassium channels may contribute to this cell death. By normalizing glutamate levels and acting on these channels, troriluzole may help prevent damage to these neurons.
Based on description provided by sponsor
At the time of submission of the application for orphan designation:
- The effects of the medicine had been evaluated in experimental models.
- No clinical trials with the medicine in patients with cystic fibrosis had been started.
More information on how potential new medicines are tested during their development is available on Authorisation of medicines.
Medicines intended for rare diseases can be granted an orphan designation during their development.
The orphan designation allows the developer to benefit from:
- scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
- market exclusivity once the medicine is on the market.
To qualify for orphan designation, a medicine must meet a number of criteria:
- it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.
EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.
The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
For more information, see:
Key facts
- Active substance
- Troriluzole hydrochloride
- Intended use
- Treatment of spinocerebellar ataxia
- Orphan designation status
- Positive
- EU designation number
- EU/3/21/2553
- Date of designation
- Sponsor
Biohaven Bioscience Ireland Limited
Update history
| Date | Update |
|---|---|
| May 2023 | The sponsorship was transferred from Biohaven Pharmaceutical Ireland DAC, Ireland to Biohaven Bioscience Ireland Limited, Ireland. |
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: