EU/3/18/2095: Orphan designation for the treatment of multiple system atrophy

Multiple system atrophy is a progressive disease of the nervous system, where nerve cells in certain areas of the brain and spinal cord gradually deteriorate, causing loss of voluntary and involuntary muscle function. This leads to symptoms such as loss of bladder control as well as shaking, rigidity and loss of muscle coordination, light-headedness due to excessive drop in blood pressure when standing up, and difficulties with speech and breathing. Some of these features are similar to those seen in Parkinson’s disease, which makes it hard to distinguish the two disorders in the early stages of the disease.

Multiple system atrophy is a long-term debilitating and life-threatening disease because of the gradual loss of muscle function and its effects on muscles used for breathing.

At the time of designation, multiple system atrophy affected less than 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 16,000 people¹, 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).

At the time of designation, there were no satisfactory methods authorised in the EU for the treatment of multiple system atrophy. Different treatments were used to relieve the symptoms of the disease, such as beta blockers and vasopressin for the treatment of hypotension (low blood pressure) and anticholinergic medicines to treat bladder problems. Parkinson medicines such as levodopa are not effective in treating the Parkinson-type symptoms of multiple system atrophy.

In patients with multiple system atrophy, a protein called alpha‑synuclein does not work properly and builds up in the brain and spinal cord forming deposits and damaging nerve cells.

This medicine is intended to work as a vaccine and stimulate the immune system (the body’s natural defences) to produce antibodies against alpha-synuclein deposits, so that the deposits are broken down and cleared from the body. This is expected to improve the symptoms of the disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with multiple system atrophy had yet been started.

At the time of submission, the medicine was not authorised anywhere in the EU for multiple system atrophy 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 18 October 2018 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.