The US Food and Drug Administration (FDA) has approved earlier on 10 March an innovative treatment for high-risk neuroblastoma, a cancer that originates from immature nerve cells and occurs primarily in children and adolescents. Unitixin is the name of the new drug, marketed by United Pharmaceuticals Inc. It is an antibody that will be used in combination with other treatment modalities such as standard chemotherapy, radiation and surgery for patients that have achieved at least a partial response to prior frontline combination therapy.

High-risk neuroblastoma patients have approximately 40-50% change of long term survival and this cancer is responsible for a substantial share of childhood cancer related deaths. A new therapeutic option for this aggressive cancer is certainly a good news.

Drug development for specific childhood cancers has been largely neglected by the pharmaceutical industry. Children with cancer are routinely treated with drugs that were originally approved as far back as the 1950s and 1960s. Over the past 20 years only two other drugs have been approved for use in children with cancer (clofarabine and L-asparaginase, both for treatment Acute Lymphoblastic Leukaemia) and most of the drugs currently in clinical practice are used off-label.

There has been a growing debate among key stakeholders on either side of the Atlantic on how the situation can be improved and more effective incentives can be enacted. It is generally agreed that the EU paediatric regulation, in force since 2007, has failed to boost drug development programmes for children with cancer. Oncology drug development is still largely driven by adult indications and most companies can obtain a waiver from the regulatory authorities on the basis that the same disease does not occur in children (often referred to as “class waiver”).

It has been argued that the outcome would improve, if better incentives were available. Indeed this idea appears to be supported by Unitixin’s approval as the company was rewarded with an FDA priority review voucher, which could be used for another drug in their pipeline or sold to another company.

This scheme was enacted into law in 2012 with the Creating Hope Act, which confers the right to a priority review voucher for any company that obtains marketing approval for a treatment against a rare paediatric disease, including childhood cancer. Unlike earlier schemes, this voucher is fully transferable and the first two indeed have been sold by the originating companies in multi-million dollar deals, indicating that this legislation might indeed create a strong financial incentive.

This piece of legislation is now under review by the US congress and supporters are seeking to re-authorize it permanently as Advancing Hope Act. Whether this bid will be successful in the long and it will actually stimulate new drug development for childhood cancer and other rare paediatric diseases, it remains to be seen. Only time will tell.

This blog post was written by Cesare Spadoni, Sr Director Business Development of Auxiliis

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