EU/3/14/1292 - orphan designation for treatment of AL amyloidosis
carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (miridesap)
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2019 on request of the sponsor.
On 29 July 2014, orphan designation (EU/3/14/1292) was granted by the European Commission to GlaxoSmithKline Trading Services Limited, Ireland, for carboxy pyrrolidine hexanoyl pyrrolidine carboxylate for the treatment of AL amyloidosis.
This medicine is now known as miridesap.
AL amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in tissues and organs such as the kidneys, liver, gut, heart and nerves.
In AL amyloidosis, the deposits come from proteins (called immunoglobulin light chains) produced in excess by malfunctioning white blood cells in the bone marrow. These deposits also contain serum amyloid P (SAP), a protein normally found in blood.
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 damage to organs, particularly the heart and kidneys.
At the time of designation, AL amyloidosis affected approximately 1.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 56,000 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 511,100,000 (Eurostat 2014).
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 complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow) was used in a small group of newly diagnosed patients.
Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (CPHPC) is expected to work by attaching to and removing the SAP protein that normally circulates in the blood.
CPHPC is to be given before treatment with another medicine, an antibody that attaches to the remaining SAP found in the amyloid deposits. Because CPHPC removes SAP from the blood, the antibody can only attach to SAP in the amyloid deposits. This is expected to stimulate the immune system to remove the unwanted material and help reduce the amyloid deposits that cause damage to the organs.
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 CPHPC in patients with AL amyloidosis were ongoing.
At the time of submission, CPHPC was not authorised anywhere in the EU for AL amyloidosis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 June 2014 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
- carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (miridesap)
- Intended use
- Treatment of AL amyloidosis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1292
- Date of designation
- Sponsor
GlaxoSmithKline Trading Services Limited
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: