EU/3/19/2221 - orphan designation for treatment of soft tissue sarcoma

camsirubicin

Overview

On 13 November 2019, orphan designation EU/3/19/2221 was granted by the European Commission to Monopar Therapeutics S.A.R.L, France, for camsirubicin for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcoma is a type of cancer that affects the soft, supportive tissues of the body. It can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect organs. Patients with soft tissue sarcoma do not usually have symptoms in the early stages of the disease. First symptoms appear when the tumour grows large enough to cause swelling and pain.

Soft tissue sarcoma is a long-term debilitating and life-threatening disease, particularly when the cancer has spread to other parts of the body.

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

At the time of designation, soft tissue sarcoma affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 181,000 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. This represents a population of 518,400,000 (Eurostat 2019).

What treatments are available?

At the time of designation, the main treatment for early-stage soft tissue sarcoma was surgery. For large sarcomas, surgery was usually followed by radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) to kill any cancer cells that were left behind. Several medicines were authorised in the EU for the treatment of soft tissue sarcoma, including doxorubicin.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma because evidence from studies in patients suggest it can be given for longer without damaging the heart than a similar authorised treatment, doxorubicin, and this may improve response.

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?

The medicine is similar to an existing cancer medicine, doxorubicin, which has been shown to be effective in treating soft-tissue sarcoma. It works by interfering with the DNA within cells, preventing them from making more copies of DNA and making proteins. This means that the sarcoma cells cannot divide and eventually die. In contrast to doxorubicin, which can only be given in limited doses because it also damages heart muscle, camsirubicin has a slightly different structure and its effects on the heart are expected to be less damaging.

What is the stage of development of this medicine?

The effects of camsirubicin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with camsirubicin in patients with soft tissue sarcoma were ongoing.

At the time of submission, camsirubicin was not authorised anywhere in the EU for the treatment of soft tissue sarcoma. Orphan designation of camsirubicin had been granted in the United States for the treatment of soft tissue sarcoma.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 10 October 2019, 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/3/19/2221: Public summary of opinion on orphan designation: Camsirubicin for the treatment of soft tissue sarcoma

Adopted Reference Number: EMADOC-628903358-1569

Key facts

• Active substance:
  camsirubicin
• Intended use:
  Treatment of soft tissue sarcoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2221
• Date of designation:
  13/11/2019
• Sponsor:

  Monopar Therapeutics SARL; 12 rue Anselme; 93400 Saint-Ouen; France; Tel: [phone]; E-mail: [e-mail]

Review of designation

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