EU/3/17/1892 - orphan designation for treatment of spinal cord injury

Oxymetazoline hydrochloride

Overview

On 17 July 2017, orphan designation (EU/3/17/1892) was granted by the European Commission to RDD Pharma Limited, United Kingdom, for oxymetazoline hydrochloride for the treatment of spinal cord injury.

What is spinal cord injury?

The spinal cord can be injured through accidents or by internal causes such as tumours or bleeding within the spine putting pressure on the spinal cord. Injury to the spinal cord can damage the nerves that run through the cord and that branch out from it. This can stop the flow of nerve impulses between the brain and the body, resulting in loss of feeling, paralysis and even death, depending upon the severity of the injury and where it is located. Patients may also experience loss of control of bladder or bowels, breathing difficulties, blood clots in veins and lungs, and recurring infections.

Spinal cord injury is a debilitating and life-threatening disease, because it can cause paralysis of the arms and legs and reduces life expectancy.

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

At the time of designation, spinal cord injury affected approximately 4.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 22,000 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).

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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 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of designation, methylprednisolone (a steroid) was authorised for the treatment of spinal cord injury in some countries in the EU. Methylprednisolone reduces the inflammation and pressure on the spinal cord that can occur after it is damaged. Patients with spinal cord injury could also have decompression surgery to reduce the pressure on the spine. Other medicines or procedures were used to manage various symptoms such as spasticity (painful muscle spasm) and incontinence.

The sponsor has provided sufficient information to show that oxymetazoline hydrochloride might be of significant benefit for patients with spinal cord injury because early studies indicate that it can reduce episodes of faecal incontinence in patients already receiving standard care. 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?

Some patients with spinal cord injury lose control of the anal sphincter (the ring of muscles that hold the anus closed) and so suffer from faecal incontinence. Oxymetazoline hydrochloride is a medicine that has been approved in the EU for many years for the treatment of nasal congestion (blocked nose). It has a relatively long-lasting action on targets called alpha adrenoceptors, which are found in various locations including the muscles of the anal sphincter. When oxymetazoline is applied locally to the area of the anus, its action is expected to stimulate these muscles to contract more tightly, reducing the risk of faecal incontinence.

What is the stage of development of this medicine?

The effects of oxymetazoline hydrochloride 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 spinal cord injury were ongoing.

At the time of submission, oxymetazoline hydrochloride was not authorised anywhere in the EU for spinal cord injury 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.

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/3/17/1892: Public summary of opinion on orphan designation: Oxymetazoline hydrochloride for the treatment of spinal cord injury

Reference Number: EMA/404401/2017

Key facts

• Active substance:
  Oxymetazoline hydrochloride
• Intended use:
  Treatment of spinal cord injury
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1892
• Date of designation:
  17/07/2017
• Sponsor:

  RDD Pharma Limited; Norfolk House; 4 Station Road; Saint Ives PE27 5AF; United Kingdom; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.