Refusal of orphan designation for treatment of fibromyalgia

naltrexone

Overview

On 7 November 2019, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for naltrexone for the treatment of fibromyalgia. A negative decision C(2019)8451 was issued by the European Commission on 16 December 2019.

The sponsor applied for orphan designation on the basis of the seriousness of the disease and insufficient returns on investment, as well as an assumption of potential benefit over currently available methods of treatment. The COMP confirmed that fibromyalgia is a seriously debilitating condition due to widespread pain that can be associated with depression and anxiety. The Committee also confirmed that there are no satisfactory methods of treatment authorised in the EU and that the medicine could provide benefits to patients with fibromyalgia. 

The negative opinion was based on the following reasons:

• The sponsor did not demonstrate that marketing the medicine in the EU is unlikely to generate sufficient return to justify the necessary investment. The sponsor did not adequately justify their view that orphan designation would reduce the costs of development, thereby providing sufficient return on investment.

Requests for designation as an orphan medicinal product are made for investigational products. Absence of orphan designation does not preclude the development of this product, including its use in clinical trials. A marketing authorisation can still be obtained if quality, safety and efficacy are demonstrated. 

Public summary of negative opinion for orphan designation of naltrexone for the treatment of fibromyalgia (Nov 2019)

Reference Number: EMADOC-628903358-1680

Key facts

• Active substance:
  naltrexone
• Intended use:
  Treatment of fibromyalgia
• Orphan designation status:
  Negative
• EU designation number:
  -
• Date of refusal of designation:
  07/11/2020
• Sponsor:

  Able AB; Vänge-Källberga 62; 74020 Vänge, Sweden; Tel: [phone]; E-mail: [e-mail]