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Orphan designation

On 29 January 2010, orphan designation (EU/3/09/711) was granted by the European Commission to NeuroNova AB, Sweden, for recombinant human vascular endothelial growth factor for the treatment of amyotrophic lateral sclerosis.

In March 2014, NeuroNova AB changed name to Newron Sweden AB.

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

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where the nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate. This causes the muscles under their control to weaken and waste away, leading to paralysis. The symptoms of ALS vary from patient to patient, depending on which muscles weaken first, and include tripping up, falling over, loss of control of hand and arm movement, difficulty speaking, swallowing and breathing, persistent tiredness, twitching and cramping. ALS usually starts in mid-life. Men are about one-and-a-half times more likely to develop the disease than women.

ALS is a debilitating disease, due to muscle deterioration, and a life-threatening disease because it affects the muscles that are used to breathe.

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

At the time of designation, ALS affected between 0.8 and 1 in 10,000 people in the European Union (EU). This was equivalent to a total of between 41,000 and 51,000 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. 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 was one medicine called riluzole authorised for ALS in the EU.
The sponsor has provided sufficient information to show that recombinant human vascular endothelial growth factor might be of significant benefit for patients with ALS because it works in a different way to existing treatment and early studies in experimental models indicate that it might improve the treatment of patients with the disease. 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?

Recombinant human vascular endothelial growth factor is a copy of a natural substance called vascular endothelial growth factor (VEGF). In the body, VEGF stimulates the development of blood vessels. The product is expected to be injected directly into the nervous system (in the ventricles of the brain), where it is expected to stimulate the growth of blood vessels to ensure better blood supply to the nerve cells, thus helping with their survival. The product is also expected to support the growth of new nerve cells.

What is the stage of development of this medicine?

The effects of recombinant human vascular endothelial growth factor have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the designated product in patients with ALS had been started.

At the time of submission, recombinant human vascular endothelial growth factor was not authorised anywhere in the EU for ALS 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 5 November 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.

Key facts

Product details for <p>Recombinant human vascular endothelial growth factor</p>
Active substanceRecombinant human vascular endothelial growth factor
Medicine Name
Disease/conditionTreatment of amyotrophic lateral sclerosis
Date of decision29/01/2010
Orphan decision numberEU/3/09/711

Review of designation

Sponsor’s contact details:

Newron Sweden AB
Fiskartorpsvagen 15 A-D
114 33 Stockholm
Tel. +46 8 786 09 00
Fax +46 8 786 09 22

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.