EU/3/22/2646 - orphan designation for treatment of ornithine transcarbamylase deficiency
mRNA encoding modified human ornithine transcarbamylase
Orphan
Human
Overview
This medicine was designated as an orphan medicine for the treatment of ornithine transcarbamylase deficiency in the European Union on 18 July 2022.
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:
Patients with ornithine transcarbamylase (OTC) deficiency lack OTC, one of the liver enzymes needed for the breakdown and removal of nitrogen in the body, resulting in high blood levels of ammonia. This medicine contains messenger RNA (mRNA) with instructions that liver cells need to make the missing OTC enzyme. The messenger RNA is enclosed in fatty particles, which prevent its breakdown in the bloodstream and help deliver it to the liver cells which make OTC. When given by infusion (drip) into a vein, the medicine is expected to allow liver cells to produce enough OTC to reduce ammonia levels and the symptoms associated with it.
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.
- Clinical trials with the medicine in patients with ornithine transcarbamylase deficiency were ongoing.
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
- mRNA encoding modified human ornithine transcarbamylase
- Intended use
- Treatment of ornithine transcarbamylase deficiency
- Orphan designation status
- Positive
- EU designation number
- EU/3/22/2646
- Date of designation
- Sponsor
Arcturus Therapeutics Europe B.V.
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: