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Orphan designation

On 22 February 2018, orphan designation (EU/3/18/1972) was granted by the European Commission to CATS Consultants GmbH, Germany, for 1-[[[4-(4-fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride (also known as AL3818 and anlotinib) for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcoma is a 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 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 191,000 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, the main treatments for soft tissue sarcoma were surgery and chemotherapy (medicines to treat cancer). Radiotherapy (treatment with radiation) was also used. Several medicines have been authorised in the EU for the treatment of soft tissue sarcoma.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma because early studies in some types of advanced sarcoma suggest improved effects with this medicine compared with authorised medicines. 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 belongs to a class of cancer medicines called tyrosine kinase inhibitors, and blocks targets called VEGF receptors on the cancer cells. These receptors are very important for the development of new blood vessels that supply blood to the cancer. By blocking VEGF receptors, it is expected that the medicine will reduce the supply of blood to the cancer and stop it from growing and spreading.

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, clinical trials with the medicine in patients with soft tissue sarcoma were ongoing.

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

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>1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride</p>
Active substance1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride
Medicine Name
Disease/conditionTreatment of soft tissue sarcoma
Date of decision22/02/2018
Orphan decision numberEU/3/18/1972

Review of designation

Sponsor’s contact details

CATS Consultants GmbH
Ussenried 7
87463 Dietmannsried
Tel. +49 8374 480

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.