EU/3/16/1608

On 17 February 2016, orphan designation (EU/3/16/1608) was granted by the European Commission to Amyndas Pharmaceuticals S.A., Greece, for S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide) for the treatment of C3 glomerulopathy.

What is C3 glomerulopathy?

C3 glomerulopathy is a condition in which a protein of the immune system (known as C3) accumulates in the kidneys, damaging them and impairing their function. The condition is caused by over-activation of part of the immune system and can be either genetic or acquired.

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.

What is the estimated number of patients affected by the condition?

At the time of designation, C3 glomerulopathy affected approximately 1.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 72,000 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).

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*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 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of orphan designation, no satisfactory treatments for C3 glomerulopathy were authorised in the EU. Because the disease involves the body’s immune system, immunosuppressant medicines were usually used. Other treatments included plasmapheresis (a blood purification procedure), plasma infusion and antiplatelet agents. Patients developing end-stage kidney disease received dialysis and kidney transplantation.

How is this medicine expected to work?

The medicine works by attaching to the C3 protein. This protein is part of a group of proteins known as the ‘complement system’, which normally helps the immune system to fight infections but which in patients with C3 glomerulopathy causes damage to the kidneys. By attaching to this protein, the medicine is expected to inactivate it, thereby slowing down the damage to the kidneys.

What is the stage of development of this medicine?

The effects of the medicine 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 C3 glomerulopathy had been started.

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 21 January 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.