SA033 is a novel treatment of cancers in liver. It is the first drug candidate that DBP has developed using its first-in-class technology BeloGal®, which enables direct targeting of a drug to the selected organ, which in the case of SA-033 means that doxorubicin is targeted directly to the liver.
Doxorubicin is one of the most well-known and proven anticancer drugs and it is the active pharmaceutical ingredient used in the SA-033 formulation. Doxorubicin is packed into a BeloGal® technology-based carrier and programmed for delivery to the liver. Following simple intravenous administration and safe transport of the formulation to its destination organ, doxorubicin is released and exerts its potent local therapeutic effect without harming healthy organs.
Treatment with SA-033 is therefore designed to significantly increase the success of liver cancer treatment and dramatically reduce the systemic (whole body) side effects of chemotherapy. For this reason, SA-033 belongs to a new generation of anticancer drugs. Double Bond Pharmaceutical AB (DBP) has received Orphan Drug Designation from the European Medicines Agency for SA-033 for the treatment of hepatoblastoma (HB), a primary malignant liver tumor and the most common liver cancer in children. The etiology of HB is unknown, but it is more common in low birth weight children and has also been associated with Beckwith-Wiedemann syndrome and familial adenomatous polyposis. The five-year survival rate after diagnosis of HB is approximately 63%, and thus there is an urgent need for more effective and safer therapy for these children.
The Orphan Drug Designation status for SA-033 received by DBP grants ten years of market exclusivity for the indication and various types of support from the EMA to further facilitate and accelerate the development of the product.