EU/3/19/2226: Orphan designation for the treatment of osteosarcoma

Osteosarcoma is a type of cancer that affects bone-forming cells. It usually affects the growing bone and occurs mainly in teenagers and young adults. Although it can develop in any bone in the body, it most often starts in the bones around the knee joint in the area of bone with the fastest growth. The most common symptom is pain, which may be worse at night. Symptoms also depend on where the cancer has spread to. The most common site of spread for this cancer is the lungs.

Osteosarcoma is a long-term debilitating and life-threatening disease that may require patients to undergo limb amputation and can shorten life.

At the time of designation, osteosarcoma affected approximately 2.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 145,000 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 osteosarcoma were surgery and chemotherapy (medicines to treat cancer). The goal of surgery was to remove cancerous bone while preserving as much skeletal function as possible. Chemotherapy was normally used to shrink the tumour before surgery and also after surgery to stop any remaining cancer cells from spreading. Chemotherapy medicines authorised in the EU for this condition included doxorubicin, cisplatin, methotrexate and ifosfamide. 

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with osteosarcoma because laboratory studies show that the medicine may be more effective at slowing the growth of osteosarcoma than authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine contains a form of doxorubicin, a cancer medicine that has been used for many years to treat cancers, including osteosarcoma. It works by interfering with the DNA in cells, preventing them from making more copies of DNA and making proteins. This means that cancer cells cannot divide and eventually die. In this medicine, the doxorubicin has been combined with hydroxybisphosphonate, a substance that is taken up by bones. This is expected to concentrate the medicine in bone tissue, increasing the amount of doxorubicin that can act on osteosarcoma cells.

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission of the application for orphan designation, no clinical trials with this medicine in patients with osteosarcoma had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of osteosarcoma or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 7 November 2019, recommending the granting of this designation.

• 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.