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Orphan designation

On 22 February 2018, orphan designation (EU/3/18/1981) was granted by the European Commission to Midatech Pharma Plc, United Kingdom, for mertansine functionalised gold nanoconjugate for the treatment of hepatocellular carcinoma.

What is hepatocellular carcinoma?

Hepatocellular carcinoma is a primary cancer of the liver (a cancer that starts in the liver, rather than one that has spread to the liver from elsewhere in the body). It is more common in men than in women, and occurs mostly in people who have liver scarring (cirrhosis), or after hepatitis B or C infection. Features of the disease include yellow skin, pain and swelling in the belly, easy bruising, weight loss, weakness, loss of appetite and nausea (feeling sick).

Hepatocellular carcinoma is a long-term debilitating and life threatening condition, with patients surviving on average for a few months or years after diagnosis.

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

At the time of designation, hepatocellular carcinoma affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 78,000 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 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, some patients with early stage hepatocellular carcinoma were treated with surgery to remove part of the liver, liver transplantation or radiofrequency ablation (directing heat and electricity through a needle to destroy cancer cells). Chemotherapy (medicines to treat cancer) was used if surgery was not possible or the disease had spread to other parts of the body (metastatic disease). Nexavar (sorafenib) was authorised in the EU for use in hepatocellular carcinoma.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients, with early laboratory studies showing that it blocks tumour growth more efficiently than the currently authorised product. 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?

Mertansine functionalised gold nanoconjugate is expected to work by targeting cancer cells and disrupting cell growth. By not allowing the cancer cells to divide and multiply, it is expected to slow down the growth and spread of the cancer.

A component of this medicine, mertansine, is a cytotoxic (cell killing) agent that had been tried as a cancer medicine in the past. This current product contains some additional components to help it target cancer cells more efficiently.

What is the stage of development of this medicine?

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 the medicine in patients with hepatocellular carcinoma had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for hepatocellular carcinoma 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 18 January 2018 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>Mertansine functionalised gold nanoconjugate</p>
Active substanceMertansine functionalised gold nanoconjugate
Medicine Name
Disease/conditionTreatment of hepatocellular carcinoma
Date of decision22/02/2018
Orphan decision numberEU/3/18/1981

Review of designation

Sponsor’s contact details

Midatech Pharma Plc - United Kingdom
65 Innovation Drive
Milton Park
OX14 4RQ
United Kingdom
Tel. 44 (0)1235 888300

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.