EU/3/06/393 - orphan designation for treatment of chronic granulomatous disease

autologous CD34+ cells transduced with retroviral vector containing the human gp91 (phox) gene

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2007 on request of the sponsor.

On 13 July 2006, orphan designation (EU/3/06/393) was granted by the European Commission to Vision 7 GmbH, Germany, for autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene for the treatment of chronic granulomatous disease.

What is chronic granulomatous disease?

Chronic granulomatous disease (CGD) is a diverse group of hereditary diseases in which certain cells of the immune system (body's natural defence against pathogens such as bacteria and viruses, and disease), known as neutrophils and monocytes have difficulty forming the reactive oxygen compounds (most importantly, the superoxide radical) used to kill certain ingested pathogens. Patients with CGD suffer from severe and recurrent bacterial and fungal infections, as their immune system cannot fight them. CGD is a life-threatening condition.

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

At the time of designation chronic granulomatous disease affected less than 1 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 46,000 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

The standard treatment for CGD includes anti-microbial prophylaxis, immunotherapy with interferon alpha (IFN-?) and aggressive early treatment of infections. Intravenous antibiotics/anti-fungal agents are the usual treatment for serious infections. Associated abscesses should be completely surgically drained as soon as possible. At the time of submission of the application for orphan drug designation, medicinal products to treat CGD were authorised in the Community.
Autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene might be of potential significant benefit for the treatment of chronic granulomatous disease because it might restore the normal function of immune system. This 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?

Autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene product is a so-called somatic cell therapy product. Patients' own (autologous) immature blood cells are isolated from the patients, and the genetic material of the cells is modified to express gp91 (phox), the missing gene in CGD. Cells are then given back to the patients and as they express gp91 they are expected to eliminate microorganisms in the same way as cells of the normal immune system of healthy individuals.

What is the stage of development of this medicine?

The evaluation of the effects of autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene in experimental models was ongoing.

At the time of submission of the application for orphan designation, clinical trials in patients with chronic granulomatous disease were ongoing.
Autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene was not authorised anywhere worldwide for chronic granulomatous disease or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 12 July 2006 a positive opinion recommending the grant of the above-mentioned 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.

EU/3/06/393: Public summary of positive opinion for orphan designation of autologous CD34+ cells transfected with retroviral vector containing the human gp91 (phox) gene for the treatment of chronic granulomatous diseas...

Adopted Reference Number: EMEA/COMP/278975/2006

Key facts

• Active substance:
  autologous CD34+ cells transduced with retroviral vector containing the human gp91 (phox) gene
• Intended use:
  Treatment of chronic granulomatous disease
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/393
• Date of designation:
  13/07/2006
• Sponsor:

  Vision 7 GmbH; Eiermarkt 3c; D-30938 Burgwedel; Germany; Telephone: [phone]; Telefax: [phone]