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.
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.
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 is based 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).
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.
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.
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.
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.
CATS Consultants GmbH
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: