EU/3/20/2300 - orphan designation for treatment of non-traumatic subarachnoid haemorrhage
fasudil hydrochloride
OrphanHuman
Overview
On 27 July 2020, orphan designation EU/3/20/2300 was granted by the European Commission to Aneuryst (Ireland) Limited, Ireland, for fasudil hydrochloride for the treatment of non-traumatic subarachnoid haemorrhage.
Non-traumatic subarachnoid haemorrhage is bleeding into the subarachnoid space (the space between two membranes that surround the brain). The bleeding is not due to an injury to the head but is usually caused by the bursting of a weakened blood-vessel wall in the brain (a cerebral aneurysm). It is a form of stroke, and the bleeding increases pressure on the brain and damages surrounding brain tissue. In addition, blood vessels near the site of the aneurysm go into spasm (vasospasm), reducing the supply of oxygen and essential nutrients to brain cells from the blood. Patients may develop seizures (fits) and other complications including heart problems.
Non-traumatic subarachnoid haemorrhage is life threatening and debilitating because of brain damage, impairment of brain functions and heart problems.
At the time of designation, non-traumatic subarachnoid haemorrhage affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 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 affected by the condition 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).
Treatment of subarachnoid haemorrhage involves surgery or a procedure using a catheter to close or block off the bleeding blood vessel. At the time of designation, nimodipine, a medicine that relaxes blood vessels and lowers blood pressure, was authorised in the EU for the prevention and treatment of complications due to vasospasm following subarachnoid haemorrhage.
The sponsor has provided sufficient information to show that the fasudil hydrochloride might be of significant benefit for patients with non-traumatic subarachnoid haemorrhage. Early data suggest that patients treated with the medicine would have reduced occurrence of vasospasms and improved symptoms compared with nimodipine.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Fasudil hydrochloride blocks an enzyme called Rho kinase which is involved in the contraction of smooth muscles, such as those in brain arteries. By blocking Rho kinase, the medicine relaxes brain arteries which are abnormally constricted in patients who have had a subarachnoid haemorrhage. This is expected to ease the supply of oxygen and essential nutrients to brain cells from the blood and reduce the symptoms of the disease.
The effects of fasudil hydrochloride 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 non-traumatic subarachnoid haemorrhage had finished.
Fasudil hydrochloride is authorised in Japan and China for the treatment of subarachnoid haemorrhage.
At the time of submission, fasudil hydrochloride was not authorised anywhere in the EU for the treatment of non-traumatic subarachnoid haemorrhage. Orphan designation of fasudil hydrochloride had been granted in the United States for the condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 18 June 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
- fasudil hydrochloride
- Intended use
- Treatment of non-traumatic subarachnoid haemorrhage
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2300
- Date of designation
- Sponsor
Inniscarra
Main Street, Rathcoole
Dublin
Ireland
Tel: + 12126711068
E-mail: info@aneuryst.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:
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: