EU/3/13/1138 - orphan designation for treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy

copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2020 on request of the Sponsor.

On 27 June 2013, orphan designation (EU/3/13/1138) was granted by the European Commission to MorEx Development Partners LLP, United Kingdom, for copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin for the treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy.

The sponsorship was transferred to Regintel Limited, Ireland, in June 2019 and subsequently to Turnkey Pharmaconsulting Ireland Limited, Ireland, in July 2020.

What is squamous-cell carcinoma of the head and neck?

Squamous-cell carcinoma of the head and neck is a type of cancer that starts in the 'squamous cells' lining the mouth, nose, throat or larynx (voice box). This type of cancer is often associated with alcohol and tobacco use, and may also be associated with local viral infection. Radiotherapy, the use of radiation to kill cancer cells, is used in about two thirds of patients with this cancer.

Squamous-cell carcinoma of the head and neck is a long-term debilitating and life-threatening disease that is associated with poor overall survival.

What is the estimated number of patients affected by the condition?

At the time of designation, squamous cell carcinoma of the head and neck in patients undergoing radiotherapy affected approximately 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 190,000 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).

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*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. At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, treatment of patients with squamous-cell carcinoma of the head and neck depended on the site and stage of development of their cancer. Surgery was widely used where possible to remove the tumour, followed by radiotherapy and chemotherapy (medicines to treat cancer), while a number of other medicines were authorised for use in this disease.

The sponsor has provided sufficient information to show that copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin might be of significant benefit for patients with squamous-cell carcinoma of the head and neck because early studies in experimental models suggest that, when used together with radiotherapy, it might offer better control over the growth of the tumour than radiotherapy alone. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Most of the damage to cells caused by treatment with radiation is not due to the direct effect of the radiation on the cells, but to the formation of 'free radicals' that react with the oxygen circulating between cells, producing very toxic molecules called 'reactive oxygen species' (ROS). These ROS are the cause of cell death. As the blood supply to cancer cells is generally poor they tend to have low levels of intracellular oxygen, which makes them less responsive to treatment with radiation.

This medicine is expected to work by diffusing into tumour tissue and acting as oxygen molecules, thereby increasing the effect of radiation on cancer cell. In addition, the copper in the medicine when exposed to the radiation is expected to emit electrons, which are highly damaging to surrounding cancer cells.

What is the stage of development of this medicine?

The effects of copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with squamous-cell carcinoma of the head and neck had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for treatment of squamous-cell carcinoma of the head and neck 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 15 May 2013 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.

EU/3/13/1138: Public summary of opinion on orphan designation: Copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin for the treatment of squamous cell carcinoma of the head...

Reference Number: EMA/COMP/309838/2013 Rev. 1

Key facts

• Active substance:
  copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin
• Intended use:
  Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/13/1138
• Date of designation:
  27/06/2013
• Sponsor:

  Turnkey PharmaConsulting Ireland Limited; Ellerman House ; Cratloe Wood ; Cratloe V95 X925; County Clare ; Ireland ; Tel: [phone] ; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.