Developed by InnoCare Pharma, ICP-192 is a:

potent and selective pan-FGFR inhibitor that we are developing for the treatment of various types of solid tumors [such as] cholangiocarcinoma with FGFR2 fusions and urothelial cancer with FGFR2/3 genetic alterations.  We developed ICP-192 with a unique structure to achieve enhanced anti-tumor efficacy while limiting in vivo drug exposure.

The Orphan Drug designation comes following data presented from a Phase 1/2a clinical trial evaluating ICP-192 in 12 patients with FGF or FGFR gene mutations. During the trial, patients received anywhere from 2-16mg ICP-192 daily. The research shows that:

• ICP-192 had an overall response rate of 33.3%.
• Of the patients who responded, 1 achieved complete response and 3 achieved partial responses. The remaining 7 patients had a stable disease state.
• Altogether, ICP-192 offered a disease control rate of 91.7% and promoted anti tumor activity.
• ICP-192 was relatively safe and well-tolerated. Currently, the maximum-tolerated dose limit has not been discovered.
• Some side effects have occurred while using ICP-192. These include increased aspartate aminotransferase, alanine aminotransferise, and blood uric acid; diarrhea; and high levels of calcium, phosphate, and triglycerides in the blood.

Orphan Drug status is granted to drugs or biologics intended to treat patients with rare conditions. In the United States, “rare” conditions affect 200,000 people or less. Alongside this status, InnoCare also receives increased regulatory assistance, fee waivers, tax credits, and 7 years market exclusivity once accepted.

Cholangiocarcinoma (Bile Duct Cancer)

Bile duct cancer, or cholangiocarcinoma, occurs in the bile ducts, tubes which connect the liver to the gallbladder and small intestine. Normally, these tubes carry bile between them. If cholangiocarcinoma occurs in bile ducts within the liver, it is called intrahepatic. Alternately, distal cholangiocarcinoma occurs in the bile ducts near the small intestine, while hilar cholangiocarcinoma occurs in the bile ducts outside of the liver. Risk factors include age (50+), having a liver parasite, smoking, having primary sclerosing cholangitis, or having chronic or congenital liver issues.

Unfortunately, cholangiocarcinoma is associated with poor prognosis and poor outcomes. This is especially true as the cancer is often asymptomatic in early stages. Many patients do not discover that they have cholangiocarcinoma until the cancer has metastasized and spread. Symptoms of cholangiocarcinoma include:

• Abdominal pain
• Extreme fatigue
• Pruritus (itchy skin)
• Jaundice (yellowing of the skin and eyes)
• Unintended weight loss
• Dark urine and light colored stool

Symptoms include:

• Pain in the abdomen
• Fatigue
• Itchy skin
• Jaundice
• White-colored stool
• Unintentional weight loss