EU/3/15/1506 - orphan designation for prevention of scarring post glaucoma filtration surgery

antisense oligonucleotide directed against TGF-β2 mRNA

Overview

On 19 June 2015, orphan designation (EU/3/15/1506) was granted by the European Commission to Isarna Therapeutics GmbH, Germany, for antisense oligonucleotide directed against TGF-β2 mRNA for the prevention of scarring post glaucoma filtration surgery.

What is scarring post glaucoma filtration surgery?

Glaucoma is a disease in which the pressure in the eye rises because the fluid inside the eye cannot drain away properly. The pressure affects the optic nerve (the nerve that sends signals from the eye to the brain) and can cause loss of vision. Glaucoma can be treated using 'filtration surgery', whereby the surgeon makes a small hole in the sclera (the white of the eye), to create a drainage channel, so that the fluid that has built up in the eye can drain away. After surgery, as part of the body's normal healing process, specialised cells called fibroblasts build up scar tissue which helps to repair the wound. However, scarring can sometimes be excessive and seal the channel up, causing the pressure inside the eye to build up again.

Scarring post glaucoma filtration surgery is a long-term debilitating condition because it may lead to loss of vision.

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of patients at risk of scarring post glaucoma filtration surgery was estimated to be approximately 1.9 people in 10,000 in the European Union (EU). This was equivalent to a total of around 97,000 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 at risk of developing 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 512,900,000 (Eurostat 2015).

What methods of prevention are available?

At the time of designation, no satisfactory methods were authorised in the EU for the prevention of scarring post glaucoma filtration surgery. Medicines that block and kill fibroblasts, such as mitomycin C or 5-fluorouracil (which are also used to treat cancer), were applied to the eye during surgery to slow down the formation of scar tissue and prevent the channel from sealing up.

How is this medicine expected to work?

This medicine is an 'anti-sense oligonucleotide', a very short piece of synthetic genetic material. This anti-sense oligonucleotide specifically attaches to a type of genetic material (called the 'sense RNA') responsible for producing the TGF-?2 protein. This protein is found in high levels in the eye of glaucoma patients and it is thought to promote scarring. When given by injection into the eye, the medicine is expected to block the production of excess TGF-?2 protein, thus preventing scarring after glaucoma filtration surgery and so stopping the pressure in the eye from building up again.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with scarring post glaucoma filtration surgery had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for scarring post glaucoma filtration surgery 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 13 May 2015 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/3/15/1506: Public summary of opinion on orphan designation: Antisense oligonucleotide directed against TGF-β2 mRNA for the prevention of scarring post glaucoma filtration surgery

Adopted Reference Number: EMA/COMP/348492/2015

Key facts

• Active substance:
  antisense oligonucleotide directed against TGF-β2 mRNA
• Medicine name:
  -
• Intended use:
  Prevention of scarring post glaucoma filtration surgery
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/15/1506
• Date of designation:
  19/06/2015
• Sponsor:

  Isarna Therapeutics GmbH; Leopoldstr. 254-256; D-80807 Munich; Germany; 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.