EU/3/21/2490 - orphan designation for treatment of lecithin-cholesterol acyltransferase deficiency
recombinant human apolipoprotein A-I
OrphanHuman
Overview
This medicine was designated as an orphan medicine for the treatment of lecithin-cholesterol acyltransferase deficiency in the European Union on 20 August 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:
Lecithin-cholesterol acyltransferase deficiency results in low circulating levels of cholesterol-containing HDL particles which normally transport cholesterol to the liver for elimination. This results in an accumulation of lipids and cholesterol in tissues, mostly in the cornea, red blood cells and kidneys. This medicine, also known as CER-001, contains replacement HDL-like particles containing human apolipoprotein A-I (produced by a method known as 'recombinant DNA technology’) in a form that can collect and transport cholesterol. This medicine is expected to facilitate the removal and elimination of accumulated excess cholesterol and lipids from peripheral tissues, including kidneys, corneas and red blood cells, hence improving or stabilising symptoms associated with the disease such as renal function, corneal opacification and anemia.
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 lecithin-cholesterol acyltransferase 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
- recombinant human apolipoprotein A-I
- Intended use
- Treatment of lecithin-cholesterol acyltransferase deficiency
- Orphan designation status
- Positive
- EU designation number
- EU/3/21/2490
- Date of designation
- Sponsor
Abionyx Pharma
33-43 Avenue Georges Pompidou
31130 Balma
France
E-mail: infos@abyonix.com
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: