EU/3/14/1415 - orphan designation for treatment of placental insufficiency
adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter
OrphanHuman
Overview
On 15 January 2015, orphan designation (EU/3/14/1415) was granted by the European Commission to Magnus Invention Management Ltd, United Kingdom, for adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter for the treatment of placental insufficiency.
Placental insufficiency is a condition in pregnancy in which the placenta, the spongy structure in the womb that nourishes the developing fetus and supplies it with oxygen from the mother's blood, does not develop properly. As a result, the baby cannot develop normally, leading to restricted growth, abnormalities of heart and lungs, and a risk of stillbirth. Babies often have to be delivered prematurely, increasing the risk of breathing problems and other severe consequences such as bleeding into the brain, damage to the gut and infections of the blood. Placental insufficiency therefore can have long-term debilitating and life-threatening consequences for the children of affected women.
At the time of designation, placental insufficiency affected approximately 3.3 in 10,000 people per year in the European Union (EU). This was equivalent to a total of around 169,000 people per year*, and is below the ceiling for orphan designation. 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
At the time of application, no satisfactory methods of treatment for placental insufficiency were authorised in the EU.
Early on in the development of a normal pregnancy, placental cells alter the mother's blood vessels supplying the womb in order to provide sufficient blood supply to the placenta to nourish the growing fetus. In pregnancies with placental insufficiency, these changes to the blood vessels do not occur to a sufficient extent, causing inadequate blood supply to the womb and the placenta.
This medicine is expected to increase the blood supply to the womb and placenta by dilating (widening) and developing blood vessels. The medicine contains parts of a virus that has been modified so that it contains the gene for a natural substance called vascular endothelial growth factor (VEGF). In the body, VEGF stimulates the development of blood vessels and causes them to widen, increasing blood flow. The medicine is expected to be injected directly into the blood vessels in the womb that supply the placenta, where the virus will be able to enter the cells and cause a temporary increase in the amount of VEGF they produce.
The type of virus used in this medicine ('adeno-associated virus') has been modified so that although it can enter cells it cannot spread or reproduce itself.
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 this medicine in patients with placental insufficiency had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for placental insufficiency 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 11 December 2014 recommending the granting of this designation.
- 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
- Active substance
- adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter
- Intended use
- Treatment of placental insufficiency
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1415
- Date of designation
- Sponsor
Magnus Invention Management Ltd
26 Red Lion Square
London WC1R 4RG
United Kingdom
Tel. +44 (0)20 3282 7567
E-mail: enquiries@magnuslifescience.co.uk
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: