EU/3/20/2354 - orphan designation for treatment of microvillus inclusion disease
Alisitol, retinol palmitate, zinc gluconate
OrphanHuman
Overview
On 13 November 2020, orphan designation EU/3/20/2354 was granted by the European Commission to Vanessa Research Magyarorszag Kft., Hungary, for alisitol, retinol palmitate, zinc gluconate (also known as Shylicine) for the treatment of microvillus inclusion disease.
Microvillus inclusion disease is an inherited disease in which the gut does not absorb nutrients from food, causing malnutrition, dehydration and affecting the normal balance of fluids and salts. This leads to severe, watery diarrhoea, weight loss and eventually problems with the liver and normal growth and development.
Microvillus inclusion disease is a long-term debilitating disease that may be life-threatening due to persistent and treatment-resistant diarrhoea requiring feeding by a drip into a vein (parenteral nutrition).
At the time of designation, microvillus inclusion disease affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of microvillus inclusion disease. Patients were normally treated with parenteral nutrition. In severe cases intestinal transplantation might be used.
Large volumes of water are normally secreted into the intestine, but most of this water is then absorbed before reaching the large intestine. Diarrhea occurs when secretion of water into the intestine exceeds absorption. Patients with microvillus inclusion disease lack special proteins called ‘channels’ to absorb salt particles from the intestine to the cells into the intestine.
Alisitol blocks the ability of special proteins called ‘chloride channels’ to carry chloride ions out of cells into the intestine and blocks secretion into the intestine. This will reduce the amount of fluid secreted into the intestine.
Retinol palmitate promotes absorption of nutrients and fluids in the gut by restoring normal structure and function of intestinal cells.
Zinc gluconate is expected to work by increasing the effects of retinol palmitate and alisitol.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with microvillus inclusion disease were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of microvillus inclusion disease or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 8 October 2020, 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
- Alisitol, retinol palmitate, zinc gluconate
- Intended use
- Treatment of microvillus inclusion disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2354
- Date of designation
- Sponsor
Vanessa Research Spain S.L.
Update history
| Date | Update |
|---|---|
| October 2023 | The sponsorship was transferred from Vanessa Research Magyarorszag Kft. to Vanessa Research Spain S.L. |
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: