On 17 October 2019, orphan designation EU/3/19/2207 was granted by the European Commission to Boyd Consultants Limited, Ireland, for 4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide (also known HM30181A) for the treatment of soft tissue sarcoma.
The sponsor’s address was updated in September 2021
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 cancer 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 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 238,000 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 518,400,000 (Eurostat 2019).
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. Early data showed improvements in angiosarcoma (sarcoma affecting blood vessels) when patients were treated with the medicine plus the cancer medicine, paclitaxel, given by mouth. Giving paclitaxel this way may reduce its side effects, so the combination could be of benefit in elderly patients who might be unfit for standard chemotherapy. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine blocks the effect of P-glycoprotein, a protein in the body that helps remove foreign substances from cells. It is expected that the medicine will allow the cancer medicine paclitaxel to work when given by mouth, helping it pass through cells in the gut and then into other cells such as cancer cells. This is expected to allow paclitaxel by mouth to enter cancer cells in sufficient quantity to slow down the growth of sarcomas and cause fewer side effects than paclitaxel given by infusion into a vein.
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 the treatment of soft tissue sarcoma. Orphan designation had been granted in the United States for angiosarcoma.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 12 September 2019, 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.
Boyd Consultants Limited
This medicine is now known as encequidar.
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:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: