EU/3/11/926 - orphan designation for treatment of primary membranoproliferative glomerulonephritis
recombinant human minibody against complement component C5
OrphanHuman
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Overview
On 27 October 2011, orphan designation (EU/3/11/926) was granted by the European Commission to Adienne S.r.l, Italy, for recombinant human minibody against complement component C5 for the treatment of primary membranoproliferative glomerulonephritis.
In January 2014, ADIENNE S.r.l. changed name to ADIENNE S.r.l.S.U.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2019 on request of the Sponsor.
Primary membranoproliferative glomerulonephritis is a kidney disease in which parts of the glomeruli, (small filtering units in the kidneys which filter waste products from the blood and start urine production), are damaged.
The damage is caused by the body's immune system attacking the glomeruli which leads to impairment of blood filtering, causing leakage of protein into the urine. Some patients have swelling in various parts of their body due to the large amount of protein that has leaked into the urine (nephrotic syndrome) while others may develop high blood pressure and have blood and protein in the urine (nephritic syndrome).
Primary membranoproliferative glomerulonephritis is a debilitating disease that is long lasting and may be life threatening because it can lead to nephrotic syndrome and complete failure of kidney function (end-stage kidney disease).
At the time of designation, primary membranoproliferative glomerulonephritis affected approximately 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 81,000 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).
At the time of orphan designation there were no satisfactory treatments authorised in the EU for this condition. Because the disease involves the body's immune system, immunosuppressant medicines were usually used. Other treatments used include plasmapheresis (a blood purification procedure), plasma infusion and antiplatelet agents. Patients developing end-stage kidney disease received dialysis and kidney transplantation.
The part of the immune system involved in damaging the glomeruli is called the complement system, which is a group of proteins in the blood that can be activated to attack cells recognised as 'foreign'.
This medicine is a small antibody (minibody) designed to target and attach to an important protein in the complement system called C5. By attaching to C5, the medicine is expected to block the chain of reactions that damages the glomeruli.
The medicine is produced by a method known as 'recombinant DNA technology': it is made by a cell that has received a gene (DNA), which makes it able to produce the medicine.
The effects of recombinant human minibody against complement component C5 have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with primary membranoproliferative glomerulonephritis had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for primary membranoproliferative glomerulonephritis 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 16 September 2011 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
- recombinant human minibody against complement component C5
- Intended use
- Treatment of primary membranoproliferative glomerulonephritis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/11/926
- Date of designation
- Sponsor
ADIENNE S.r.l.S.U.
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: