EU/3/18/1989

C3 glomerulopathy is a condition in which a protein of the immune system (body's natural defences) known as C3 builds up in the kidneys, damaging them and impairing their function.

Patients with C3 glomerulopathy usually have signs of kidney problems, such as blood or protein in urine, high blood pressure, blurred vision and swelling in the hands and feet.

C3 glomerulopathy is life threatening and debilitating in the long term because it can lead to kidney failure.

At the time of designation, C3 glomerulopathy affected approximately 1.1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 57,000 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 517,400,000 (Eurostat 2018).

At the time of orphan designation, no satisfactory treatments were authorised in the EU for C3 glomerulopathy. Because the disease involves the body's immune system, immunosuppressant medicines (that reduce the activity of the immune system) were usually used. Other treatments included plasmapheresis (a procedure to remove unwanted substances from plasma, the liquid part of the blood), infusion of healthy plasma, and medicines to reduce blood pressure and protein in the urine. Some patients developing end-stage kidney disease received dialysis and kidney transplantation.

The damaging build-up of the C3 protein in kidneys of patients with C3 glomerulopathy mostly results from overactivity of an enzyme called C3 convertase. This medicine blocks another protein called factor D, which is involved in the production of C3 convertase. By blocking factor D and hence production of C3 convertase, the medicine is expected to reduce the build-up of the C3 protein and relieve the symptoms of the disease.

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 C3 glomerulopathy were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for C3 glomerulopathy 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 15 February 2018 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.