EU/3/17/1893 - orphan designation for treatment of anti-MAG neuropathy
Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside
OrphanHuman
Overview
On 17 July 2017, orphan designation (EU/3/17/1893) was granted by the European Commission to SFL Regulatory Affairs Consulting Ltd, United Kingdom, for polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside for the treatment of anti-MAG neuropathy.
The sponsorship was transferred to SFL Regulatory Services GmbH, Austria, in December 2018 and subsequently to Pharma Gateway AB, Sweden, in September 2020.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2021 on request of the Sponsor.
Anti-MAG neuropathy is a progressive disease of the peripheral nerves (nerves outside the brain and spinal cord). In this disease, the body's immune (defence) system produces antibodies that incorrectly target and destroy a natural substance called myelin-associated glycoprotein (MAG). MAG is an important part of the protective sheath around the nerve cells in the body. The condition typically occurs in people aged above 50 years and is more common in men than in women. Patients with the condition lose sensation first in their fingers and toes and go on to develop muscle weakness, lack of coordination, tremors and nerve pain. As the condition worsens, patients need nursing support and become wheelchair bound.
Anti-MAG neuropathy is debilitating in the long term because of increasing inability to control muscle movement and loss of sensation. The condition is not however considered life-threatening.
At the time of designation, anti-MAG neuropathy affected approximately 2.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 119,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 515,700,000 (Eurostat 2017).
At the time of designation, no satisfactory methods were authorised for the treatment of anti-MAG neuropathy. Treatment included medicines to reduce the activity of the immune system, and procedures to remove antibodies from the blood.
This medicine attaches to MAG antibodies and stops them attaching to MAG. By neutralising MAG antibodies in this way, the medicine is expected to prevent the immune system from attacking MAG. This is expected to stop damage to the protective sheath around nerve cells and prevent progression of anti-MAG neuropathy.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with anti-MAG neuropathy had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for anti-MAG neuropathy 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 15 June 2017 recommending the granting of this designation.
- 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.
Key facts
- Active substance
- Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside
- Intended use
- Treatment of anti-MAG neuropathy
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1893
- Date of designation
- Sponsor
Pharma Gateway AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: