Overview

On 21 March 2016, orphan designation (EU/3/16/1633) was granted by the European Commission to Diamond BioPharm Limited, United Kingdom for fosbretabulin tromethamine for the treatment of gastro-entero-pancreatic neuroendocrine tumours.

The sponsorship was transferred to Diamond ROC EOOD, Bulgaria, in March 2019.

The sponsorship was transferred to Diamond Pharma Services Ireland Limited in February 2020.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2021 on request of the Sponsor.

 

Gastro-entero-pancreatic neuroendocrine tumours (GEP-NETs) are tumours that arise from neuroendocrine cells in the gut. These cells release hormones that control various functions of the digestive system. The symptoms of GEP-NETs depend on where in the gut the tumour is growing and on whether it produces excess hormones. Often by the time of diagnosis the tumours have spread to other organs such as the liver.

GEP-NETs are debilitating as they often produce excess hormones that may cause severe symptoms. They are life-threatening if they spread to other organs in the body.

At the time of designation, GEP-NETs affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 178,000 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).

*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 513,700,000 (Eurostat 2016).

At the time of designation, several products were authorised in the EU for treating GEP-NETs, including lanreotide and octreotide for the management of symptoms, everolimus and sunitinib.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with GEP-NETs because early data showed positive responses in previously treated patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Fosbretabulin tromethamine is a medicine that disrupts the cells of blood vessels supplying the tumour, disrupting small structures called microtubules within the cells which allow these cells to divide. By disrupting these blood vessels, it is expected to cause the death of tumour cells by cutting off their blood supply, and thereby slow down the progression of the disease.

The effects of fosbretabulin tromethamine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with GEP-NETs were ongoing.

At the time of submission, Fosbretabulin tromethamine was not authorised anywhere in the EU for GEP-NETs. Orphan designation had been granted in the EU for ovarian cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 February 2016 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.

EU/3/16/1633: Public summary of opinion on orphan designation: Fosbretabulin tromethamine for the treatment of gastro-entero-pancreatic neuroendocrine tumours

AdoptedReference Number: EMA/COMP/172769/2016Summary:

Orphan designation

English (EN) (213.11 KB - PDF)

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Key facts

Active substance
Fosbretabulin tromethamine
Intended use
Treatment of gastro-entero-pancreatic neuroendocrine tumours
Orphan designation status
Withdrawn
EU designation number
EU/3/16/1633
Date of designation
Sponsor

Diamond Pharma Services Ireland Limited 
Coliemore House
Coliemore Road
Dalkey
Co. Dublin
Ireland 
Tel. 02039119410
E-mail: mgraham@diamondpharmaservices.com

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:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

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:

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