EU/3/16/1801 - orphan designation for treatment of amyotrophic lateral sclerosis
Ibudilast
OrphanHuman
Overview
On 12 December 2016, orphan designation (EU/3/16/1801) was granted by the European Commission to MediciNova (Europe) Limited, United Kingdom, for ibudilast for the treatment of amyotrophic lateral sclerosis.
The sponsorship was transferred to Medicinova Europe GmbH, Germany, in October 2019.
Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. The exact causes are unknown but are believed to include genetic and environmental factors. The symptoms of ALS depend on which muscles weaken first, and include loss of balance, loss of control of hand and arm movement, and difficulty speaking, swallowing and breathing. ALS usually starts in mid-life and men are more likely to develop the disease than women.
ALS is a debilitating and life-threatening disease because of the gradual loss of function and its paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.
At the time of designation, ALS affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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 513,700,000 (Eurostat 2016).
At the time of designation, riluzole was authorised in the EU to treat ALS. Patients also received supportive treatment, such as physiotherapy and breathing support, to relieve the symptoms of the disease.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ALS, with early studies showing breathing and muscle function did not worsen so quickly when ibudilast is given in combination with riluzole. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The exact way ibudilast works in ALS is unclear but it is known to block the action of enzymes called phosphodiesterases (PDEs) and of a substance called macrophage migration inhibiting factor (MIF), which are involved in regulating certain functions of brain cells. By blocking MIF and PDEs, the medicine is expected to suppress the production of inflammatory molecules and to increase the production of 'protective' molecules. In addition, ibudilast reduces activity of glial cells (a type of brain cell thought to be involved in the development of ALS). Together, these actions are expected to improve the symptoms of ALS.
The effects of ibudilast 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 ALS were ongoing.
At the time of submission, ibudilast was not authorised anywhere in the EU for ALS. Orphan designation of the medicine had been granted in the United States for ALS.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 November 2016 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
- Ibudilast
- Intended use
- Treatment of amyotrophic lateral sclerosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1801
- Date of designation
- Sponsor
Medicinova Europe GmbH
Leopoldstrasse 16
Schwabing Freimann
80802 Munich
Germany
E-mail: inquiries@medicinova.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: