Overview

On 18 November 2016, orphan designation (EU/3/16/1778) was granted by the European Commission to Pharmalink AB, Sweden, for budesonide for the treatment of primary IgA nephropathy.

In January 2018, Pharmalink AB changed name to Calliditas Therapeutics AB.

The sponsor’s address was updated in July 2021.

The medicinal product (for orphan indication in case of several orphan indications or type II variation) has been authorised in the EU as Kinpeygo since 15 July 2022.

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 isbased 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.

EU/3/16/1778: Public summary of positive opinion for orphan designation of budesonide for the treatment of primary IgA nephropathy

Key facts

Active substance
Budesonide
Intended use
Treatment of primary IgA nephropathy
Orphan designation status
Positive
EU designation number
EU/3/16/1778
Date of designation
Sponsor

STADA Arzneimittel AG
Stadastrasse 2-18
Dortelweil
61118 Bad Vilbel
Hassia
Germany
E-mail: info@stada.de

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Kinpeygo at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report.

Update history

DateUpdate
September 2022The sponsorship was transferred from Calliditas Therapeutics AB, Sweden to STADA Arzneimittel AG, Germany in September 2022.

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:

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:

How useful do you find this page?