EU/3/08/543 - orphan designation for treatment of amyotrophic lateral sclerosis

sarsasapogenin

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products on 23 July 2012 on request of the sponsor.

On 8 May 2008, orphan designation (EU/3/08/543) was granted by the European Commission to Phytopharm plc, United Kingdom for sarsasapogenin for the treatment of amyotrophic lateral sclerosis.

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis is a progressive disease of the nervous system. Amyotrophic lateral sclerosis occurs when specific nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate. The loss of these so-called motor neurones causes the muscles under their control to weaken and waste away, leading to paralysis.

Amyotrophic lateral sclerosis varies from patient to patient, depending on which muscles weaken first. Symptoms may include tripping and falling, loss of motor control in hands and arms, difficulty in speaking, swallowing or breathing, persistent fatigue, and twitching and cramping. Amyotrophic lateral sclerosis strikes in mid-life. Men are about one-and-a-half times more likely to have the disease than women. Amyotrophic lateral sclerosis is chronically debilitating and life-threatening.

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

At the time of designation, amyotrophic lateral sclerosis affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 50,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the 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 based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,800,000 (Eurostat 2008). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

A medicinal product called riluzole was authorised for the condition in the Community at the time of submission of the application for orphan drug designation. Sarsasapogenin might be of potential significant benefit for the treatment of amyotrophic lateral sclerosis. The benefit 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?

Neurotrophic factors are proteins that improve the survival and function of nerve cells. Although the mechanism of action of sarsasapogenin in amyotrophic lateral sclerosis is not fully understood, it is thought that it increases neurotrophic factors and mimics their effects in the nervous system, and thereby improves the symptoms of the disease.

What is the stage of development of this medicine?

The effects of sarsasapogenin were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with amyotrophic lateral sclerosis were planned.

Sarsasapogenin was not authorised anywhere worldwide for treatment of amyotrophic lateral sclerosis at the time of submission.

Orphan designation of sarsasapogenin was granted in the United States for the treatment of amyotrophic lateral sclerosis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 March 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;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) 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.

EU/3/08/543: Public summary of positive opinion for orphan designation of sarsasapogenin for the treatment of amyotrophic lateral sclerosis

Adopted Reference Number: EMEA/COMP/202469/2008

Key facts

• Active substance:
  sarsasapogenin
• Intended use:
  Treatment of amyotrophic lateral sclerosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/543
• Date of designation:
  08/05/2008
• Sponsor:

  Phytopharm plc; Lakeview House; 2 Lakeview Court; Ermine Business Park; Huntingdon; Cambridgeshire PE29 6UA; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]