EU/3/18/1984 - orphan designation for treatment of primary IgA nephropathy

recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2

Overview

On 22 February 2018, orphan designation (EU/3/18/1984) was granted by the European Commission to Omeros London Limited, United Kingdom, for recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 (also known as OMS721) for the treatment of primary IgA nephropathy.

The sponsorship was transferred to Omeros Ireland Limited, Ireland, in January 2021.

What is 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 build-up damages the glomeruli, causing leakage of 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.

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

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 207,000 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).

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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 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, no satisfactory methods were authorised for treating primary IgA nephropathy. Patients were treated with ACE inhibitors or angiotensin receptor blockers to lower blood pressure, and with medicines to suppress the immune system, such as corticosteroids, ciclosporin or cyclophosphamide. As the disease worsens, kidney dialysis and kidney transplant may be needed.

How is this medicine expected to work?

This medicine is made up of a monoclonal antibody (a type of protein) that has been designed to attach to and block an enzyme called mannan-binding lectin serine protease 2 (MASP-2). This enzyme is involved in activating proteins in the 'complement system', which is part of the immune system. The complement system plays a role in inflammation of the kidneys in primary IgA nephropathy. By blocking MASP-2, the medicine is expected to reduce the immune response that causes the symptoms of the disease.

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, clinical trials with the medicine in patients with primary IgA nephropathy were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for primary IgA nephropathy. Orphan designation of the medicine had been granted in the United States for primary IgA nephropathy.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 January 2018 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.

EU/3/18/1984: Public summary of opinion on orphan designation: Recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 for the treatment of primary IgA nephropathy

Adopted Reference Number: EMA/64972/2018

Key facts

• Active substance:
  recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2
• Intended use:
  Treatment of primary IgA nephropathy
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/1984
• Date of designation:
  22/02/2018
• Sponsor:

  Ormond Building; 31-36 Ormond Quay Upper; Dublin 7 ; D07 EE37; Co. Dublin; Ireland; Email: [e-mail]