EU/3/16/1778: Orphan designation for the treatment of primary IgA nephropathy

Primary IgA nephropathy is a disease caused by the immune system (the body's natural defences) producing a faulty version of an antibody called immunoglobulin A (IgA), which builds up in small blood vessels in the kidney, called glomeruli, that filter the blood. This damages the glomeruli and makes them leak blood and protein into the urine.

Primary IgA nephropathy is a long-term debilitating and life-threatening disease because the kidneys gradually stop working properly and eventually fail, requiring dialysis or a kidney transplant.

At the time of designation, primary IgA nephropathy affected approximately 4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 205,000 people¹, 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).

¹Disclaimer: 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).

At the time of submission of the application for orphan drug designation, no satisfactory methods were authorised for primary IgA nephropathy. Patients were often treated with medicines to suppress the immune system, such as corticosteroids, ciclosporin or cyclophosphamide. Patients at risk of developing kidney failure were given medicines to lower blood pressure, such as ACE inhibitors or angiotensin receptor blockers, to reduce stress on the kidneys. As the disease worsens, kidney dialysis and kidney transplant may be needed.

Budesonide is a well-known type of medicine called a corticosteroid. Corticosteroids have a wide range of effects that suppress the immune system. This medicine is designed to be released when it reaches the intestine, where it is expected to reduce the production of faulty IgA antibodies from immune tissue in the intestine. This is expected to decrease their build-up in the kidneys and so reduce damage to the kidneys.

At the time of submission of the application for orphan designation, clinical trials with budesonide in patients with primary IgA nephropathy were ongoing.

At the time of submission, budesonide was authorised in the EU for treatment of inflammatory conditions such as asthma, Crohn's disease and ulcerative colitis.

At the time of submission, budesonide was not authorised anywhere in the EU for primary IgA nephropathy. Orphan designation of budesonide had been granted in the US for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 October 2016 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.