EU/3/06/399

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Orphan designation

On 28 August 2006, orphan designation (EU/3/06/399) was granted by the European Commission to ROP Pharma AB, Sweden, for mecasermin rinfabate for the prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age.
The sponsor changed name to Premacure AB in September 2007.

What is the retinopathy of prematurity?

Children that are born preterm are deprived of their natural environment in the womb, and they lack important factors normally provided to the unborn child, such as proteins, cellular growth factors and cytokines. The last weeks of a full-term pregnancy are also important for the growth of the eye, in particular for the formation of blood vessels supplying blood to the retina (the area at the back of the eye that receives light and sends pictures of what the eye sees to the brain). In some premature infants, the normal growth of the retinal vessels stops, and abnormal new vessels begin to grow. Therefore, the oxygen supply to the retina is limited. The formation of abnormal vessels is accompanied by the production of scar tissue. In some cases this can result in visual impairment and in extreme cases in blindness.

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

At the time of designation the population at risk of developing retinopathy of prematurity in neonates of less than 32 weeks of gestational age was approximately 1.2 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 55,000 people.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What methods of prevention are available?

At the time of the submission of orphan drug designation application, there were no medicinal products approved for the prevention of condition in the Community. The treatment consisted of laser or cryotherapy (local treatment with “ice”) to close the abnormal new vessels and to limit the scar tissue.

How is this medicine expected to work?

One factor that stimulates cell growth and helps cells to develop special features for specific functions (specialization) is insulin-like growth factor 1 (IGF-1). A lack of it may lead to abnormal vessel growth in retina that is typical for retinopathy of prematurity. IGFBP-3 is a protein that binds to IGF-I and regulates the availability and activity of IGF-1. Mecasermin rinfabate mimics the effects of this natural protein complex (IGF-1/IGFBP-3) in the bloodstream and is able to stay in the body for a longer period. Therefore, the product is expected to promote the normal physiological development of the eye in premature neonates.

What is the stage of development of this medicine?

The effects of the medicinal product were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with condition were initiated.

Mecasermin rinfabate was not authorised anywhere worldwide for prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age or designated as orphan medicinal product for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 24 July 2006 a positive opinion recommending the grant of the above-mentioned 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Mecasermin rinfabate</p>
Active substanceMecasermin rinfabate
Medicine Name
Disease/conditionPrevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age
Date of decision28/08/2006
OutcomePositive
Orphan decision numberEU/3/06/399

Review of designation

Sponsor’s contact details:

Premacure AB
Uppsala Science Park
SE-751 83 Uppsala
Sweden
Telephone: +46 70 650 28 52
Telefax: +46 18 572 350
E-mail: info@premacure.com

Patients’ associations contact points:

Pro Retina Deutschland e.V.
Vaalser Straße 108
52074 Aachen
Germany
Telephone: +49 241 87 00 18
Telefax: +49 241 87 39 61
E-mail: Pro-Retina@t-online.de

BRPS - British Retinitis Pigmentosa Society
PO Box 350
Buckingham
MK18 1GZ
United Kingdom
Telephone: +44 1280 82 13 34
Telefax: +44 1280 81 59 00
E-mail: info@brps.org.uk

IRRP - Association Information Recherche sur la Rétinite Pigmentaire
53 Rue de la République
30160 Besseges
France
Telephone: +33 4 66 25 13 54
Telefax: +33 4 66 25 13 54
E-mail: contact@irrp.asso.fr