Refusal of orphan designation for treatment of autosomal dominant polycystic kidney disease

sodium ascorbate

menadione sodium bisulfite

Overview

On 21 July 2014, the Committee for Orphan Medicinal Products (COMP) adopted a negative opinion on the orphan designation application for sodium ascorbate and menadione sodium bisulfite for the treatment of autosomal dominant polycystic kidney disease. A negative decision was issued by the European Commission on 11 December 2014.

The sponsor applied for orphan designation on the basis of the seriousness and the rarity of the condition.

The negative opinion was based on the following reasons:

• The sponsor calculated the prevalence of autosomal dominant polycystic kidney disease on the basis of a limited number of publications, and the methodology used to estimate the prevalence was not adequately justified.
• The COMP noted that the prevalence of the genotype for the disease is higher than 5 in 10,000 people in the European Union and that the sponsor could not prove that the disease itself does not have a prevalence higher than this figure. 5 in 10,000 people is the maximum allowable prevalence for orphan designation.

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 opinion on orphan designation: Sodium ascorbate and menadione sodium bisulfite for the treatment of autosomal dominant polycystic kidney disease

Adopted Reference Number: EMA/COMP/736555/2014

Key facts

• Active substance:
  • sodium ascorbate
  • menadione sodium bisulfite
• Intended use:
  Treatment of autosomal dominant polycystic kidney disease
• Orphan designation status:
  Negative
• EU designation number:
  -
• Date of refusal of designation:
  11/12/2014
• Sponsor:

  JJGConsultancy Ltd; Sherston House; High Street; Evercreech; Somerset BA4 6HZ; United Kingdom; Tel. [phone]; Fax [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.