EU/3/15/1586 - orphan designation for treatment of neurotrophic keratitis

Recombinant human nerve growth factor (cenegermin)

Overview

This medicine is now known as cenegermin.

On 14 December 2015, orphan designation (EU/3/15/1586) was granted by the European Commission to Dompé farmaceutici S.p.A., Italy, for recombinant human nerve growth factor for the treatment of neurotrophic keratitis.

Recombinant human nerve growth factor has been authorised in the EU as Oxervate since 6 July 2017.

What is neurotrophic keratitis?

Neurotrophic keratitis is a condition of the cornea (the clear layer at the front of the eye) caused by damage to the trigeminal nerve, the nerve that allows the surface of the eye to feel things and which supplies growth factors and other substances needed for normal growth and repair of the eye's surface. The nerve damage results in a lack of sensitivity in the cornea, and to dryness, ulceration and scarring that interferes with vision.

Neurotrophic keratitis is a long-lasting and debilitating condition due to damage to the cornea and possible loss of sight.

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

At the time of designation, neurotrophic keratitis affected approximately 4.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 215,000 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 512,900,000 (Eurostat 2015).

What treatments are available?

No satisfactory methods were authorised for the treatment of neurotrophic keratitis in the EU at the time of designation. Management depended on the stage of the disease and included supportive measures such as eye drops to moisten and lubricate the eye, antibiotics for eye infections, and the use of protective contact lenses, as well as surgery where appropriate.

How is this medicine expected to work?

Patients with neurotrophic keratitis have lower than normal levels of growth factors that are normally supplied by the trigeminal nerve and which play an important role in the growth and survival of the cells of the cornea. The medicine is a copy of a human growth factor called nerve growth factor. When given as eye drops to patients with neurotrophic keratitis, the medicine is expected to help restore some of the normal healing processes in the eye and repair the damages to the cornea associated with the condition.

The medicine is produced by a method known as 'recombinant DNA technology': it is made by bacteria into which a gene (DNA) has been introduced that enables the bacteria to produce human nerve growth factor.

What is the stage of development of this medicine?

The effects of recombinant human nerve growth factor have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with recombinant human nerve growth factor in patients with neurotrophic keratitis were ongoing.

At the time of submission, recombinant human nerve growth factor was not authorised anywhere in the EU for neurotrophic keratitis. Orphan designation of the medicine had been granted in the United States for neurotrophic keratitis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 November 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/3/15/1586: Public summary of opinion on orphan designation: Recombinant human nerve growth factor for the treatment of neurotrophic keratitis

AdoptedReference Number: EMA/COMP/793645/2015

Key facts

• Active substance:
  Recombinant human nerve growth factor (cenegermin)
• Intended use:
  Treatment of neurotrophic keratitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/15/1586
• Date of designation:
  14/12/2015
• Sponsor:

  Dompé farmaceutici S.p.A.; Via Santa Lucia 6; 20122 Milano; Italy; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

On 19 May 2017, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/15/1586 for Oxervate (cenegermin, previously known as recombinant human nerve growth factor) as an orphan medicinal product for the treatment neurotrophic keratitis. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained.¹

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^[1]The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.