EU/3/19/2222 - orphan designation for treatment of AL amyloidosis
chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4
OrphanHuman
Overview
On 13 November 2019, orphan designation EU/3/19/2222 was granted by the European Commission to Real Regulatory Limited, Ireland, for chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4 (also known as NSC-711516 and CAEL-101) for the treatment of AL amyloidosis.
The sponsorship was transferred to Alexion Europe S.A.S., France in January 2022.
AL amyloidosis belongs to a group of diseases in which deposits of proteins (called amyloids) accumulate and cause damage in body organs. In AL amyloidosis, the deposits are made of abnormal proteins (called immunoglobulin light chains) produced in excess by malfunctioning white blood cells in the bone marrow.
In patients with AL amyloidosis, the amyloids can accumulate and cause damage in the kidneys, heart, liver, spleen, nerves, or digestive system. Symptoms of the condition vary widely depending on which organs are affected by the deposits and how much deposits have accumulated in them.
AL amyloidosis is a life-threatening and long-term debilitating condition because of the damage to organs, particularly the heart and kidneys.
At the time of designation, AL amyloidosis 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 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 518,400,000 (Eurostat 2019).
At the time of designation, no medicines were authorised in the EU for the treatment of AL amyloidosis. Patients often received treatment with medicines (chemotherapy) originally designed to treat cancers of white blood cells, in order to target the malfunctioning white blood cells. Stem cell transplantation (a procedure where the patient’s bone marrow is cleared of cells and replaced by stem cells from a donor to form new bone marrow) was used in a small group of newly diagnosed patients.
This medicine is an antibody (a type of protein) which has been designed to attach to the abnormal protein in amyloid deposits and thereby stimulate the immune system to attack the deposits. This action is expected to help reduce the deposits of amyloid, thereby preventing organ damage and improving the patient’s symptoms.
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 AL amyloidosis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for AL amyloidosis. Orphan designation of the medicine had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 10 October 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
- chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4
- Intended use
- Treatment of AL amyloidosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2222
- Date of designation
- Sponsor
Alexion Europe S.A.S.
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product it 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: