On 25 November 2009, orphan designation (EU/3/09/692) was granted by the European Commission to Biogenera srl, Italy, for 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide for the treatment of neuroblastoma.
- What is neuroblastoma?
Neuroblastoma is a cancer of nerve cells in the abdomen (tummy) and manifests itself as a lump in the abdomen or around the spine. Symptoms may include weakness, bone pain, loss of appetite and fever. Neuroblastoma is the most common solid tumour outside the brain in children. It is often present at birth but is usually diagnosed later when the cancer has spread to other parts of the body and the child begins to show symptoms of the disease.
Neuroblastoma is a life-threatening disease of childhood that is associated with poor long-term survival.
- What is the estimated number of patients affected by the condition?
At the time of designation, neuroblastoma affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 50,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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. This represents a population of 504,800,000 (Eurostat 2009).
- What treatments are available?
At the time of designation, several medicines were authorised for the treatment of neuroblastoma in the EU. Treatments for neuroblastoma included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide might be of significant benefit for patients with neuroblastoma because it works in a different way to existing treatments, and early studies in experimental models indicate that it might be used in combination with existing treatments to improve the overall outcome of patients with this condition. These assumptions 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?
16-Base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide is expected to work by attaching to and blocking the gene responsible for producing a protein called MYCN. This gene is involved in the growth, progression and spread of neuroblastomas that are particularly severe. By blocking the gene, the medicine blocks the production of the MYCN protein. This is expected to stop the neuroblastoma cells from growing and multiplying.
- 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 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide in experimental models was ongoing.
At the time of submission of the application for orphan designation, no clinical trials with the designated product in patients with neuroblastoma had been started.
At the time of submission, 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide was not authorised anywhere in the EU for neuroblastoma 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 7 October 2009 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 Community) 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.
|Name||Language||First published||Last updated|
|EU/3/09/692: Public summary of positive opinion for orphan designation of 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide for the treatment of neuroblastoma||(English only)||06/12/2009|
|Active substance||16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide|
|Disease/condition||Treatment of neuroblastoma|
|Date of decision||24/11/2009|
|Orphan decision number||EU/3/09/692|
Review of designation
Sponsor’s contact details:
Via Marconi 46
Porretta Terme 40046 (BO)
Telephone: +39 051 18899343
Telefax: +39 051 19980272
Patient associations’ contact points:
Ligue Nationale Contre le Cancer
14 Rue Corvisart
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
Macmillan Cancer Support
3 Bath Place
London EC2A 3JR
Telephone: +44 20 7696 9003
Telefax: +44 20 7696 9002
European Cancer Patient Coalition
Am Rothenanger 1b 85521
Telephone: +49 89 628 36 807
Telefax: +49 89 628 36 808