The European Commission granted marketing authorisation for United Therapeutics‘ Unituxin for the treatment of neuroblastoma in patients aged 12 months to 17 years.
In Europe, approximately 1500 patients are diagnosed with neuroblastoma, the most common cancer in children. Some patients – the low-risk and intermediate risk group – are favored by an excellent prognosis with cure rates above 90%. However, in approximately 50% of the cases, the disease is difficult to treat. These are the patients that could benefit from United Therapeutics’ new treatment.
The company was founded in 1996 by parents of a child with pulmonary arterial hypertension, a disorder characterized by constricted pulmonary arteries, that lead to oxygen deficiency. Today, the Nasdaq-listed company has a market cap of nearly $8 billion (€7,3Bn). Unituxin is the company’s fifth product on the market.
It is a monoclonal antibody that interacts specifically with a certain molecule that is highly expressed on the surface of the neuroblastoma cells. In therapy, Unituxin will be combined with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and isotretinoin. The combined treatment increased the two-year event-free survival to 66%, compared to the 48% in patients receiving isotretinoin alone. It also increased the overall survival. With the combined treatment, 80% of the patients survived for at least 3 years, compared to 67% who received isotretinoin alone. Some tough side-effects such as severe pain or eye problems have been noticed.
This new treatment may be a rare hope for the suffering children and their families. Back in March, this therapy became the first on the US market to treat children with high-risk neuroblastoma who achieved at least a partial response to prior multimodal therapy such as chemotherapy or surgery. Finally, European children can also benefit from the new therapy.