Osteosarcoma – a rare disease in need of new treatments

Osteosarcoma – a rare disease in need of new treatments

Osteosarcoma is a primary cancer bone tumor commonly diagnosed in patients with growing bones. Thus, the disease is most common in adolescents under an age of 30 and about 1000 individuals in Europe and the USA are diagnosed with the disease each year. Tremendous improvements of osteosarcoma treatment have occurred since 1960 including the introduction of chemotherapies, such as the use of high dose methotrexate in combination with folates as rescue treatment. Availability of improved treatments have changed the 5 year survival rates from 15 to about 70% in patients with a localized disease stage. However, among the ~20% of patients being diagnosed with metastatic disease, the 5 year survival rates (~20%) has not gone through a significant change during the recent decades and can be seen as an unmet medical need in the market.


Very few drugs have been approved in osteosarcoma during the last decades but two exceptions are MEPACT (Takeda) and FUSILEV (Spectrum Pharmaceuticals). MEPACT contains the active ingredient mifamurtide that kills cancer cells by producing monocytes and macrophages. The EMA approved MEPACT in 2009 for patients with high-grade non-metastatic osteosarcoma but the drug was denied an approval by the FDA (as it was claimed by the US authority to have failed to provide substantial evidence of efficacy). Moreover, the FDA approved levoleucovorin based FUSILEV in 2008. FUSILEV can be seen as an improved version of the existing drug leucovorin for the usage as a rescue agent in osteosarcoma patients treated with high-dose methotrexate. Unfortunately, these drugs have not had a large impact on the outcome for patients with metastatic disease.


It is therefore interesting to look at the clinical pipeline for what’s coming. Indeed, there is a number of early stage pipeline candidates in development for osteosarcoma (e.g. Quadramet, Cometriq and Afinitor). A majority of these are targeting patients with metastatic disease thus trying to fill this unmet need. Many of the pipeline candidates are already approved for other indications and may be developed within osteosarcoma as life cycle management strategies by extending the labels of these into other indications. Osteosarcoma is classified as a rare disease (reported prevalence of 5-7.5 / 10 000 in some regions) and in 2013-2014, ODDs were granted for two pipeline candidates, namely Advaxis’ ADXSHER2 (ODD in the US) and Eleison Pharmaceutical´s Inhaled Lipid-Complexed Cisplatin (ODD in Europe).


Will any new drug candidates be approved in the coming years and will they manage to challenge the current standard treatments and possibly extend the survival rates of patients with metastatic disease? Monocl will continue to analyze the market updates within osteosarcoma. Keep following us for market development news.

CXO and Co-founder of Monocl Software