EU/3/07/521

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Orphan designation

On 18 December 2007, orphan designation (EU/3/07/521) was granted by the European Commission to Vaxon Biotech, France, for H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt & H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt for the treatment of TERT positive non-small cell lung cancer in HLA-A2 positive patients.

What is TERT positive non-small cell lung cancer in HLA-A2 positive patients?

Non-small cell lung cancer originates in the lung and is the most common form of lung cancer, accounting for about 80% of all lung cancer cases. The disease is characterised by a tumour in the lungs which often spreads into surrounding tissues and other parts of the body causing organ failure. Non-small cell lung cancer is a very serious disease, and it is chronically debilitating and life-threatening.

TERT (telomerase reverse transcriptase) is an enzyme (a protein that speeds up the conversion of certain substances into other substances) that is often present in non-small cell lung cancer tumour cells. These cells are then called TERT positive cells. TERT is needed for tumour cells to be able to divide many times, and subsequently for the tumour to grow.

On the outer membrane of cells in the body there is a protein called major histocompatability complex that displays parts of other proteins that are present inside the cell. This is a way for the cell to communicate what is going on inside it to the rest of the body. The major histocompatibility complex is very variable between individuals: there can be great differences in its structure from person to person. One of the variants of the major histocompatibility complex that is common in Europe is called HLA-A2.

What treatments are available?

The treatment of non-small cell lung cancer depends on how advanced the disease is. If the cancer is discovered at an early stage, before it has spread outside the lungs, it can sometimes be removed surgically. Other ways of treating non-small cell lung cancer is by chemotherapy (using drugs to kill cancer cells) or by radiotherapy (using high energy rays to kill cancer cells). There were several medicinal products authorised for the treatment of non-small cell lung cancer at the time of application.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt & H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt might be of potential significant benefit for the treatment of TERT positive non-small cell lung cancer in HLA-A2 positive patients, mainly because it has a new mechanism of action, could potentially provide a safer option to current treatments and may potentially work in patients where other methods of treatment are unsuccessful. These assumptions will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

What is the stage of development of this medicine?

H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt & H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt are two peptides (short fragments of proteins) that are similar to a distinct part of TERT. The product is designed to work as a vaccine (to activate the body’s natural defence system, the immune system, against a specific disease or foreign compound) against TERT. According to the sponsor the product will trigger the immune system against the non-small cell cancer tumour cells that display TERT fragment on their surface in the major histocompatability complex variant HLA-A2, thus destroying the tumour cells with the body’s own defence system.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, TERT positive non-small cell lung cancer in HLA-A2 positive patients was considered to affect less than 4 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 200,000 persons.

*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 Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What is the stage of development of this medicinal product?

The effects of H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt & H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with TERT positive non-small cell lung cancer in HLA-A2 positive patients were ongoing.

H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt & H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt was not authorised anywhere worldwide for the treatment of TERT positive non-small cell lung cancer in HLA-A2 positive patients nor 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 8 November 2007 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

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>H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt&amp; H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt</p>
Active substanceH-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt& H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt
Medicine Name
Disease/conditionTreatment of TERT positve non-small cell lung cancer in HLA-A2 positive patients
Date of decision17/12/2007
OutcomePositive
Orphan decision numberEU/3/07/521

Review of designation

Sponsor’s contact details:

Vaxon Biotech
CIT BP 191 
3 rue de l’Arrivée 
75749 Paris Cedex 15 
France
Telephone: + 33 1 45 88 67 62
Telefax: + 33 1 45 88 67 80
E-mail: kkosmatopoulos@vaxon-biotech.com

Patients’ associations contact points:

Cancer BACUP
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 76 96 90 03 / 0808 800 1234 (freephone for UK)
Telefax: +44 20 76 96 90 02

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net

The Association of European Cancer Leagues (ECL)
c/o Belgian Federation against Cancer
Chaussée de Louvain, 479
B-1030 Brussels
Belgium
Telephone: +32 2 743 3705
Telefax: +32 2 734 9250
E-mail: chartmann@cancer.be