Senhwa Biosciences has announced the receipt of Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for Silmitasertib, a highly selective inhibitor of casein kinase 2 (CK2) to treat patients with biliary tract cancer (BTC).
Senhwa Biosciences acting CEO Mei-Hui Kuo said: “We are pleased to receive ODD for Silmitasertib for the treatment of Biliary Tract Cancer, a rare, malignant disease for which there are no effective therapies.
“ODD represents an important regulatory milestone that has the potential to expedite the clinical development of Silmitasertib, which is a potent and selective CK2 inhibitor.”
The US regulatory agency provides ODD status to those drugs and biologics meant for treatment, prevention or diagnosis of a life-threatening or chronically debilitating rare disease that has a prevalence of fewer than 200,000 people.
ODD enables some financial incentives that can support clinical development, including the potential for up to seven years of market exclusivity, in the country following approval by the regulatory agency.
BTC is a group of rare, diverse and aggressive cancers arising from the bile duct system.
BTCs are categorised into four distinct subtypes based on the tissue where the cancer originates. These subtypes are intrahepatic cholangiocarcinoma (IHCC), extrahepatic cholangiocarcinoma (EHCC), gallbladder cancer (GBC) and ampullary cancer.
In the early-stages, patients having BTC often have nonspecific symptoms which in turn can complicate and delay diagnosis.
Patients with BTC are usually diagnosed once it has already advanced or spread.
A chemotherapy regimen of gemcitabine + cisplatin forms as the first-line standard of care.
According to the firm, there are no globally accepted standards of care for locally advanced or metastatic BTC following the failure of the first line chemotherapy.
Pre-clinical studies indicate that inhibition of CK2 by Silmitasertib prevents DNA repair, induces apoptosis, and boosts the antitumor activity of gemcitabine and cisplatin.
Senhwa Biosciences added there is a need for new BTC treatment options.