EU/3/05/275

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Orphan designation

On 11 April 2005, orphan designation (EU/3/05/275) was granted by the European Commission to B. Braun Melsungen AG, Germany, for estradiol hemihydrate and progesterone for the prevention of bronchopulmonary dysplasia in premature neonates of less than 30 weeks of gestational age.

The sponsorship was transferred to Dr Frank Pohlandt, Germany, in September 2011.

What is bronchopulmonary dysplasia?

Bronchopulmonary dysplasia (BPD) is a chronic lung disease that develops in premature neonates and is associated with the infant’s low birth weight and gestational age. BPD is caused by several factors such as a lack of the protective coating of the lungs due to immaturity, bacterial infections and artificial oxygen administration to support the breathing. These factors can damage the lungs, which means that the normal functions of the lung tissue (exchange of oxygen and carbon dioxide between air and blood) are impaired. This leads to inflammation (the body’s to the injury caused to the tissue) and progressive formation of scar tissue. The patient will develop an increasing shortness of breath. Bronchopulmonary dysplasia is a chronically debilitating and life-threatening condition. 

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

At the time of designation, the number of neonates of less than 30 weeks of gestational age at risk of bronchopulmonary dysplasia was estimated to be approximately 1.3 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 60,000 people, which 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 based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What methods of prevention are available?

At the time of submission of the application for the orphan drug designation there was no preventive treatment authorised in the European Union. Corticosteroids are commonly used to prevent bronchopulmonary dysplasia in premature neonates of less than 30 weeks of gestational age as they induce maturation of the lungs. 

How is this medicine expected to work?

Estradiol hemihydrate and progesterone are able to increase the functional and structural maturation of the lungs. They are expected to prevent the development of the condition in premature neonates of less than 30 weeks of gestational age by substituting the estradiol and progesterone that the neonate would normally receive from the mother if the intrauterine life would continue. 

What is the stage of development of this medicinal product?

The effects of estradiol hemihydrate and progesterone were evaluated in experimental models. 

At the time of submission of the application for orphan designation, clinical trials in patients with bronchopulmonary dysplasia were ongoing. 

Estradiol hemihydrate and progesterone was not authorised anywhere worldwide for bronchopulmonary dysplasia or designated as orphan medicinal product elsewhere 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 3 March 2005 a positive opinion recommending the grant of the above-mentioned designation.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 March 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 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

Key facts

Product details for <p>Estradiol hemihydrate and progesterone</p>
Active substanceEstradiol hemihydrate and progesterone
Medicine Name
Disease/conditionBronchopulmonary dysplasia in premature neonates of less than 30 weeks of gestational age
Date of decision11/04/2005
OutcomePositive
Orphan decision numberEU/3/05/275

Review of designation

Sponsor’s contact details:

Dr Frank Pohlandt
Fünf-Bäume-Weg 138/1
89081 Ulm
Germany
Telephone: +49 731 381548
Telefax: +49 731 381548
E-mail: frank.pohlandt@uni-ulm.de

Patients’ associations:

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.