EU/3/01/064 - orphan designation for treatment of lupus nephritis

abetimus sodium

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in November 2009 on request of the sponsor.

On, 20 November 2001 orphan designation (EU/3/01/064) was granted by the European Commission to Icon Clinical Research UK Ltd., United Kingdom, for abetimus sodium for the treatment of lupus nephritis.

The sponsorship was transferred to La Jolla Limited, United Kingdom in September 2005.

What is lupus nephritis?

Lupus nephritis is a disease characterised by inflammation of the kidney caused by systemic lupus erythematosus (SLE), a disease of the immune system. SLE affects the skin, joints, kidneys and brain. The causes of SLE are unknown. Patients with SLE produce autoantibodies. Antibodies are proteins of the immune system that normally target bacteria and viruses. Autoantibodies mistakenly attack tissues. Lupus nephritis may cause weight gain, high blood pressure, dark urine, or swelling around the eyes, legs, ankles or fingers. This inflammation can damage the internal organs. Lupus nephritis is a chronically debilitating and life-threatening condition because of complications occurring during the disease process such as chronic renal failure and infections.

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

At the time of designation lupus nephritis affected approximately 0.6 to 1.8 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 23,000 to 69,000 people.

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union. This represents a population of 377,000,000 (Eurostat 2001).

What treatments are available?

At the time of submission of the application for the orphan drug designation there were glucocorticoids and other immunosuppressive agents (compounds that reduce the activity of the immune system) authorised for the condition in the Community.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that abetimus sodium might be of potential significant benefit for the treatment of lupus nephritis.

The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Abetimus sodium is a chemically synthesised double-stranded oligodeoxyribonucleotide (oligonucleotides are the building blocks of the genetic material, DNA), which is targeted by the autoantibodies produced in patients with lupus nephritis. According to the sponsor, the medicinal product interferes with the function of B lymphocyte cells (type of immune cells) and it is expected to prevent the immune system from attacking and damaging patients' own tissues.

What is the stage of development of this medicine?

The effects of abetimus sodium were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with lupus nephritis were ongoing.

Abetimus sodium was not marketed anywhere worldwide for the treatment of lupus nephritis. Orphan designation of abetimus sodium was granted in the United States in 2000 for the treatment of lupus nephritis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2001 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 European Union) 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/3/01/064: Public summary of positive opinion for orphan designation of abetimus sodium for the treatment of lupus nephritis

Adopted Reference Number: EMEA/COMP/267/04

Key facts

• Active substance:
  abetimus sodium
• Intended use:
  Treatment of lupus nephritis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/01/064
• Date of designation:
  20/11/2001
• Sponsor:

  La Jolla Limited,; 20-22 Bedford Row,; London WC1R 4JS,; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]