EU/3/19/2134 - orphan designation for treatment of infantile neuroaxonal dystrophy
9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester
OrphanHuman
Overview
On 26 February 2019, orphan designation (EU/3/19/2134) was granted by the European Commission to FGK Representative Service GmbH, Germany for 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester (also known as RT001) for the treatment of infantile neuroaxonal dystrophy.
Infantile neuroaxonal dystrophy is a genetic disorder that mainly affects the nervous system. The condition usually starts in children before the age of 3 years, with the child’s having difficulties with movement and slowly losing vision and intellectual skills.
The disease is caused by the build-up of substances in the nerves endings throughout the brain and body, which prevents them from working properly.
Infantile neuroaxonal dystrophy is a debilitating and life-threatening disease because of its progressive symptoms. The disease usually leads to death in childhood.
At the time of designation, infantile neuroaxonal dystrophy affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 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 518,400,000 (Eurostat 2019).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of infantile neuroaxonal dystrophy. Patients received supportive treatment such as physiotherapy and medicines to relieve symptoms and improve the patient's general condition.
The medicine works by blocking production of the substances that build up in the nerves endings of patients with infantile neuroaxonal dystrophy. As a result, the medicine is expected to prevent the damages caused by their build-up in patients with the condition.
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 infantile neuroaxonal dystrophy were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for infantile neuroaxonal dystrophy. Orphan designation had been granted in the United States for PLA2G6–associated neurodegeneration.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 24 January 2019 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
- 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester
- Intended use
- Treatment of infantile neuroaxonal dystrophy
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2134
- Date of designation
- Sponsor
FGK Representative Service GmbH
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:
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: