EU/3/05/282 - orphan designation for treatment of acanthamoeba keratitis

Miltefosine

Overview

On 27 May 2005, orphan designation (EU/3/05/282) was granted by the European Commission to Obwaller Forschungs- und Entwicklungs GmbH, Austria, for miltefosine for the treatment of Acanthamoeba keratitis.

The sponsorship was transferred to Laboratoires Théa, France, in July 2007, to Orphanidis Pharma Research GmbH, Austria, in December 2008 and subsequently to Orpha-Devel Handels und Vertriebs GmbH, Austria, in December 2015.

What is Acanthamoeba keratitis?

Acanthamoeba are organisms consisting of one single cell that can cause an eye disease. These organisms live and survive in many different places such as air, soil and water environments. From these sources, they can gain access to contact-lens solutions and contact lenses. Tap water used to clean lenses is a common source of such an infection. The Acanthamoeba may be on the lens or in the cleaning solutions. If lenses are left in the eyes for long periods of time, these amoebae can multiply and cause damage to the eye (tears and ulcers).

Acanthamoeba keratitis is a chronically debilitating condition and may result in loss of sight in the infected eye.

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

At the time of designation, Acanthamoeba keratitis affected not more than 0.1 in 10,000 people in 10,000 people in the European Union (EU). This is equivalent to a total of not more than 4,700 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).

---

*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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).

What treatment are available?

No satisfactory methods exist that were authorised at the time of application.

How is this medicine expected to work?

Although it is not yet fully understood how miltefosine acts in Acanthamoeba keratitis, it seems that it might destroy the walls of the amoeba cells. Therefore, it might eradicate the amoeba (amoebicidal).

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, no clinical trials in patients with Acanthamoeba keratitis had been started.

The medicinal product was not marketed anywhere worldwide for Acanthamoeba keratitis or designated as an orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 April 2005 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 Community) 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/3/05/282: Public summary of positive opinion for orphan designation of miltefosine for the treatment of Acanthamoeba keratitis

Adopted Reference Number: EMEA/COMP/139086/2005 Rev. 4

Key facts

• Active substance:
  Miltefosine
• Intended use:
  Treatment of acanthamoeba keratitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/05/282
• Date of designation:
  27/05/2005
• Sponsor:

  Orpha-Devel Handels und Vertriebs GmbH; Wintergrasse 85/1B; 3002 Purkersdorf; Austria; Tel. [phone]; Fax [phone]; E-mail: [e-mail]