EU/3/10/825

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Orphan designation

On 17 December 2010, orphan designation (EU/3/10/825) was granted by the European Commission to Laboratoires SERB, France, for ovine anti-colchicine polyclonal antibody fragments for the treatment of colchicine poisoning.

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

What is colchicine poisoning?

Colchicine is a natural product that was originally extracted from plants. It is used mainly to treat gout (high levels of uric acid in the blood causing symptoms especially painful inflammation in the joints). Colchicine is toxic at doses that are very close to the dose used for treatment and this can lead to poisoning.

Colchicine poisoning can be intentional (mainly by suicide attempts) or happen by mistake, through a single overdose or excessive use of colchicine-containing medicines over time, particularly in patients with kidney or liver problems as they are less able to eliminate it from the body.

Colchicine poisoning is potentially life-threatening as it can rapidly lead to severe complications including cardiogenic shock (inadequate circulation of blood due to the heart’s failure to function properly), respiratory failure and multiple organ failure.

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

At the time of designation, colchicine poisoning affected less than 0.01 people in 10,000 per year in the European Union (EU). This is equivalent to a total of fewer than 500 people per year*, which was considered to be below the ceiling for orphan designation. This is based 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, there were no satisfactory methods of treatment authorised in the EU for colchicine poisoning. Depending on the stage and severity of the poisoning, the condition was treated with procedures intended to stop the body absorbing the poisonous substance from the gut and to treat the resulting complications. These include emergency procedures such as gastric lavage (stomach pumping), oxygen supplementation, fluid replacement and emergency life-support treatment.

How is this medicine expected to work?

Ovine anti-colchicine polyclonal antibody fragments are expected to be used as an antidote to colchicine. They are made from antibodies produced from sheep. An antibody is a protein that helps the body to fight infections and other diseases by attaching to and helping to destroy antigens.

The antibody fragments are expected to work by attaching to colchicine in the human body. This prevents colchicine from attaching to the protein that it normally attaches to inside the cells, redistributing it away from the cells. This prevents damage to the organs and body tissues and allows the colchicine to be eliminated through the urine.

What is the stage of development of this medicine?

The effects of anti-colchicine polyclonal antibody fragments derived from goats, but not from sheep, have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with ovine anti-colchicine polyclonal antibody fragments were planned but had not been started.

At the time of submission, ovine anti-colchicine polyclonal antibody fragments were not authorised anywhere in the EU for colchicine poisoning or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 October 2010 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>Ovine anti-colchicine polyclonal antibody fragments</p>
Active substanceOvine anti-colchicine polyclonal antibody fragments
Medicine Name
Disease/conditionTreatment of colchicine poisoning
Date of decision17/12/2010
OutcomePositive
Orphan decision numberEU/3/10/825

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

Laboratoires SERB
40 avenue George V
75008 Paris
France
Tel. +33 1 73 03 20 00
Fax +33 1 46 36 75 47
E-mail: regulatory@serb-labo.com

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.