According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, “biliary cancer” can consist of bile duct/biliary tract cancer (also known as cholangiocarcinoma) or gallbladder cancer.
Devimistat
Developed by Rafael Pharmaceuticals, Inc. (“Rafael“), devimistat:
targets enzymes that are involved in cancer cell energy metabolism and are located in [cancer cell] mitochondria.
Through this targeted treatment, devimistat works to make cancer cells more sensitive to chemotherapy. In terms of advanced biliary tract cancer, Rafael is running a Phase 1/2 clinical trial to evaluate the efficacy of devimistat in conjunction with chemotherapy versus chemotherapy alone. Since biliary cancer can be aggressive and difficult to treat, this therapy offers to fulfill an unmet need.
The drug received Orphan Drug status, which is granted to drugs or biologics intending to treat patients with rare diseases (affecting under 200,000 Americans). As a result, Rafael is privy to fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity once devimistat is approved. Outside of biliary cancer, devimistat has also received Orphan Drug designation for:
- Soft-tissue sarcoma
- Acute myeloid leukemia (AML)
- Pancreatic cancer
- Myelodysplastic syndromes (MDS)
- Burkitt lymphoma
Biliary Cancer
As described above, biliary cancer consists of cancer affecting the bile duct system. Normally, the bile duct system helps move bile through the liver, gallbladder, and small intestine. But when cancer develops, a number of health issues may occur. Biliary tract and bile duct cancer, or cholangiocarcinoma, form in the tubes which carry bile from the liver to the small intestine. The cancer can be intrahepatic (forming in bile ducts within the liver), distal (forming in bile ducts closest to the small intestine), and hilar (forming in bile ducts outside of the liver). As the name suggests, gallbladder cancer forms in the gallbladder. Risk factors for developing biliary cancer include age (50+), being obese, having diabetes, smoking, or having primary sclerosing cholangitis.
Symptoms associated with biliary cancer typically do not appear until later stages. Once symptoms appear, they include:
- Jaundice (yellowing of the skin and eyes)
- Abdominal pain
- Unintended weight loss
- Nausea and vomiting
- Appetite loss
- Pruritus (intense itchiness)
- Feeling of fullness
- Pain from metastases
- Light-colored stool or dark urine
- Fever (with no known cause)
