EU/3/08/611

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Orphan designation

On 11 February 2009, orphan designation (EU/3/08/611) was granted by the European Commission to Alfact Innovation SAS, France, for recombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein for the treatment of acute liver failure.

What is acute liver failure?

Acute liver failure is characterised by the sudden loss of the normal liver functions in a patient with a previously normal liver and without evidence of chronic liver disease. The most common first sign of liver failure is yellowing of the skin (jaundice). Acute liver failure has serious complications such as confusion, drowsiness, bruising and haemorrhages secondary to impaired blood clotting. The most common causes of acute liver failure are viral hepatitis (infectious disease that affects the liver) or toxic damage (e.g. alcohol and paracetamol). Acute liver failure is chronically debilitating and life-threatening.

What is the estimated number of patients affected by acute liver failure?

At the time of designation acute liver failure affected less than 3.5 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 161,000 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 (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

Treatment of acute liver failure depends mainly on the stage of the liver damage. In cases of advanced liver damage, liver transplantation (getting a liver from a donor) is performed.

The sponsor has provided sufficient information to show that recombinant human hepatocarcinoma-intestine-pancreas/pancreatic associated protein might be of significant benefit, as through its mechanism of action it may result on a reduction of the liver damage that this organ suffers in a situation of acute liver failure, and therefore could contribute to a better clinical outcome of patients affected by acute liver failure. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Recombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein is a protein produced in microorganisms (recombinant) derived from the human protein HIP/PAP (Hepatocarcinoma-Instestine-Pancreas /Pancreatitis Associated Protein). HIP/PAP is a protein that has shown in experiments to be able to help cells generating new cells (cell proliferation) and to protect cells from dying after being damaged (anti apoptotic effect). These functions are hoped to limit the liver damage and to improve the healing of the liver in acute liver failure.

What is the stage of development of this medicine?

The effects of recombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with acute liver failure had been started.

At the time of submission, Recombinant human hepatocarcinoma-intestine-pancreas/pancreatic associated protein for the treatment of acute liver failure ‘was not authorised anywhere in the world for acute liver failure or designated as 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 10 December 2008 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Recombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein</p>
Active substanceRecombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein
Medicine Name
Disease/conditionTreatment of acute liver failure
Date of decision10/02/2009
OutcomePositive
Orphan decision numberEU/3/08/611

Review of designation

Sponsor’s contact details:

Alfact Innovation SAS
320, rue Saint Honoré
75001 Paris
France
Telephone: + 33 1 53 40 60 51
Telefax: + 33 1 53 40 60 52
E-mail: mariau@alfactinnovation.com

Patients’ associations contact points:

British Liver Trust
Portman House
44 High Street
Ringwood
BH24 1AG
United Kingdom
Telephone: +44 14 25 46 30 80
E-mail: info@britishlivertrust.org.uk

Euroliver Foundation
Markgravestraat, 10 P.O.
B-2000 Anvers
Belgium
Telephone: +32 3 226 79 97
Telefax: +32 3 226 79 70
E-mail: euroliver@glo.be