EU/3/06/407 - orphan designation for prevention of necrotising enterocolitis

heparin-binding epidermal growth factor-like growth factor, amino acids 74-148

Overview

On 31 October 2006, orphan designation (EU/3/06/407) was granted by the European Commission to Dr Michael Moore, United Kingdom, for heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148 for the prevention of necrotizing enterocolitis.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is necrotizing enterocolitis?

Necrotizing enterocolitis is a disease that affects preterm infants. The gut and the immune system (the body's natural defence system) of these very low weight infants are immature and not as well developed as full term infants', increasing the risk of infection during the first few weeks after birth. “Necrotizing enterocolitis” means that portions of the gut (both small and large intestine) may undergo tissue death due to the infection. The symptoms may include problems with feeding, abdominal distension, and bloody stools. If the disease progresses, it can lead to peritonitis and sepsis (infection of the abdominal cavity and infection of the blood). These complications are life-threatening and due to the risk of long-term disabilities, necrotizing enterocolitis may also be chronically debilitating.

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 necrotizing enterocolitis was estimated to be approximately 1.2 people in 10,000 in the European Union (EU). This was equivalent to a total of around 56,000 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).

---

*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 468,900,000 (Eurostat 2006).

What methods of prevention are available?

The condition is treated with surgery if possible (removal of the damaged intestinal tissue). There were, however, no products authorised for the prevention of necrotizing enterocolitis in the Community at the time of submission of application for orphan drug designation.

How is this medicine expected to work?

Heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148 is a part of protein naturally occurring in the amniotic fluid (the fluid that the baby is surrounded by in the womb). This protein is known to stimulate growth and development of epithelial cells, which include the cells that line the inside of the intestines. According to the sponsor, heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148 might thus help prevent necrotizing enterocolitis in low birth weight preterm infants.

What is the stage of development of this medicine?

The evaluation of the effects of heparin-binding epidermal growth factor-like growth factor (HB EGF), amino acids 74-148 in experimental models is ongoing. At the time of submission of the application for orphan designation, no clinical trials in patients at risk of being affected by necrotizing enterocolitis were initiated.

Heparin-binding epidermal growth factor-like growth factor (HB EGF), amino acids 74-148 was not authorised anywhere worldwide for the prevention of necrotizing enterocolitis, at the time of submission. Orphan designation of heparin-binding epidermal growth factor-like growth factor (HB EGF), amino acids 74-148 was granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 September 2006 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/3/06/407: Public summary of positive opinion for orphan designation of heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148 for the prevention of necrotizing enterocolitis

Adopted Reference Number: EMEA/COMP/183997/2008 Rev. 1

Key facts

• Active substance:
  heparin-binding epidermal growth factor-like growth factor, amino acids 74-148
• Intended use:
  Prevention of necrotising enterocolitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/06/407
• Date of designation:
  31/10/2006
• Sponsor:

  Dr Michael Moore; 11 Fairway Heights; Clewborough Drive; Camberley; Surrey GU15 1NJ; United Kingdom; Tel. + 44 (0)7532 85803 / 801; Fax + 44 (0)7532 85803 / 801