EU/3/19/2169 - orphan designation for treatment of spinal muscular atrophy

reldesemtiv

Overview

On 28 June 2019, orphan designation EU/3/19/2169 was granted by the European Commission to Pharma Gateway AB, Sweden, for reldesemtiv for the treatment of spinal muscular atrophy.

What is spinal muscular atrophy?

Spinal muscular atrophy is an inherited disease usually diagnosed in the first year of life that affects the motor neurons (nerves from the brain and spinal cord that control muscle movements). Patients with the disease lack a protein called ‘survival motor neuron’ (SMN), which is essential for the normal functioning and survival of motor neurons. Without this protein, the motor neurons deteriorate and eventually die. This causes the muscles to fall into disuse, leading to muscle wasting (atrophy) and weakness.

Spinal muscular atrophy is a long-term debilitating and life-threatening disease because it causes breathing problems and muscle wasting that worsens over time.
 

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

At the time of designation, spinal muscular atrophy affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 16,000 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).

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*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).

What treatments are available?

At the time of designation, the medicine Spinraza (nusinersen) was authorised for the treatment of spinal muscular atrophy. Spinraza is given by injection into the spine. Patients also received supportive treatment to help them and their families cope with the symptoms of the disease. This included chest physiotherapy and physical aids to support muscle function, and ventilators to help with breathing.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with spinal muscular atrophy. Data from laboratory studies indicate that when used together with nusinersen this medicine could improve patients’ muscle function compared with results seen with Spinraza alone.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
 

How is this medicine expected to work?

This medicine is expected to activate voluntary muscles by attaching to a protein known as troponin, which is involved in muscle contractions. By doing so, the medicine is expected to strengthen the muscles’ response to nerve stimulation, thereby improving muscle function in patients with spinal muscular atrophy.

What is the stage of development of this medicine?

The effects of reldesemtiv have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with reldesemtiv in patients with spinal muscular atrophy had been started.

At the time of submission, reldesemtiv was not authorised anywhere in the EU for the treatment of spinal muscular atrophy. Orphan designation of reldesemtiv had been granted in the United States for spinal muscular atrophy.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a Positive opinion on 23 May 2019, 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/3/19/2169: Public summary of opinion on orphan designation: Reldesemtiv for the treatment of spinal muscular atrophy

Adopted Reference Number: EMADOC-628903358-1039 Summary:

On 28 June 2019, orphan designation EU/3/19/2169 was granted by the European Commission to Pharma Gateway AB, Sweden, for reldesemtiv for the treatment of spinal muscular atrophy.

Key facts

• Active substance:
  reldesemtiv
• Intended use:
  Treatment of spinal muscular atrophy
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2169
• Date of designation:
  28/06/2019
• Sponsor:

  Cytokinetics (Ireland) Limited