EU/3/19/2155 - orphan designation for treatment of amyotrophic lateral sclerosis
Human culture expanded autologous mesenchymal stromal cells
OrphanHuman
Overview
On 24 April 2019, orphan designation (EU/3/19/2155) was granted by the European Commission to IQVIA RDS Ireland Limited, Ireland, for human culture expanded autologous mesenchymal stromal cells (also known as lenzumestrocel) for the treatment of amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. The exact causes are unknown but are believed to include genetic and environmental factors. The symptoms of ALS depend on which muscles weaken first, and include loss of balance, loss of control of hand and arm movement, and difficulty speaking, swallowing and breathing. ALS usually starts in mid-life and men are more likely to develop the disease than women.
ALS is a debilitating and life-threatening disease because of the gradual loss of function and its paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.
At the time of designation, ALS affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 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 on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 518,400,000 (Eurostat 2019).
At the time of designation, riluzole was authorised in the EU to treat ALS. Baclofen was authorised for treating muscle symptoms which include those of ALS. Patients also received supportive treatment to relieve the symptoms of the disease, such as physiotherapy and breathing support.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ALS because preliminary results show that adding it to riluzole slowed progression of the condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine contains cells called mesenchymal stromal cells (MSCs) obtained from the patient's bone marrow and grown in a laboratory to increase their numbers. MSCs are thought to increase production of substances that prevent inflammation, protect nerve cells and reduce the activity of the immune system (the body's natural defences). Injecting the medicine into the patient's spine is expected to prevent the immune system from damaging nerve cells and so prevent worsening of the condition.
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 ALS were ongoing.
The medicine is authorised in South Korea for the treatment of ALS.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of ALS. Orphan designation of the medicine had been granted in the United States for ALS.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 21 March 2019, 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
- Human culture expanded autologous mesenchymal stromal cells
- Intended use
- Treatment of amyotrophic lateral sclerosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2155
- Date of designation
- Sponsor
Parexel International (Irl) Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation
Update history
| Date | Update |
|---|---|
| May 2022 | The sponsorship was transferred to Parexel International (Irl) Limited, Ireland in April 2022. |
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:
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.
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: