Silmitasertib, an investigative therapy for biliary tract cancer (BTC), has been awarded Orphan Drug Designation by the FDA. Researchers and the regulatory agency are hopeful that this therapy could become an effective treatment option for this patient population with a high unmet need.

BTC

Biliary tract cancer, or cholangiocarcinoma, currently has no effective treatment for any of its subtypes. The subtypes of BTC include ampullary cancer, extrahepatic cholangiocarcinoma (EHCC), intrahepatic cholangiocarcinoma (IHCC), epithelial adenocarcinoma, and gallbladder cancer (GBC). The subtypes are classified by the type of tissue the cancer originates in. The most common place for BTC to originate is in the epithelial cells, making epithelial adenocarcinoma the most common subtype. In fact nine out of every 10 patients diagnosed has epithelial adenocarcinoma.

The current standard of care for BTC is cisplatin and gemcitabine. However, there is no global standard of care when this chemotherapy combination fails.

This patient population drastically needs more options.

Silmitasertib

Silmitasertib acts as a CK2 inhibitor and targets the CK2 pathway. It is currently being studied as a treatment for many different cancers in both pediatric and adult patients.

The therapy has thus far shown to be safe and effective in humans. It’s also ideal because it’s an oral treatment patients can take from their own home. Preclinical studies have demonstrated that the therapy induces apoptosis, prevents repair of DNA, and also improves the effectiveness of cisplatin and gemcitabine.

So far 3 Phase 1 clinical trials as well as 1 Phase 2 clinical trial of the therapy have been completed. Additionally, there are 2 Phase 2 clinical trials which are ongoing.

ODD

Orphan Drug Designation is provided to treatments believed to have the potential to drastically improve outcomes for rare disease patients who currently have no or limited treatment options. Silmitasertib being granted FDA ODD demonstrates that the FDA believes in the potential of this therapy for BTC.

ODD provides many incentives to researchers in order to aid in the research and clinical development of the treatment. It also provides 7 years marketing exclusivity following FDA approval of the treatment.

You can read more about this designation and what it could mean to BTC patients here.