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Orphan designation

On 8 February 2013, orphan designation (EU/3/13/1100) was granted by the European Commission to Onclave Therapeutics Limited, Ireland, for humanised IgG1 kappa antibody against serum amyloid A and AL amyloid for the treatment of amyloid light-chain amyloidosis.

In March 2015, Onclave Therapeutics Limited changed name to Prothena Therapeutics Limited.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is amyloid light-chain amyloidosis?

Amyloid light-chain amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in body organs. In amyloid light-chain amyloidosis, the amyloids consist of components of defective proteins (called light chains) that are produced by malfunctioning cells. The condition can cause serious damage to organs such as the kidneys, liver, gut, heart and nervous system.

Amyloid light-chain amyloidosis is a long-term debilitating condition because of damage to organs such as the heart and kidneys.

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

At the time of designation, amyloid light-chain amyloidosis affected approximately 1.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 56,000 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).

*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.
At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, no medicines were authorised in the EU for the treatment of amyloid light-chain amyloidosis. Stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow) was used in a small group of newly diagnosed patients.

How is this medicine expected to work?

This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (called an antigen). When injected into the patient's vein, it is expected to attach to the light chain proteins in the amyloids, breaking them down and allowing their removal from the body. This is expected to reduce the amyloid deposits in the organs and thereby the organ damage caused by the disease.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicinal product in experimental models was ongoing.

At the time of submission, no clinical trials with the medicinal product in patients with amyloid light-chain amyloidosis had been started.

At the time of submission, the medicinal product was not authorised anywhere in the EU for amyloid light-chain amyloidosis. Orphan designation of the medicinal product had been granted in the United States for amyloid light-chain amyloidosis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2013 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.

Key facts

Product details for <p>Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid</p>
Active substanceHumanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Medicine Name
Disease/conditionTreatment of amyloid light-chain amyloidosis
Date of decision08/02/2013
Orphan decision numberEU/3/13/1100

Review of designation

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

Sponsor’s contact details

Prothena Therapeutics Limited
Adelphi Plaza
Upper George's Street
Dun Laoghaire
Co. Dublin A96 T927
Tel. +353 1 236 2500
Tel. +1 650 837 8537
Fax +353 1 686 5675

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.