Intrahepatic Cholangiocarcinoma (iCCA): Causes, Symptoms, and New Treatment

Cholangiocarcinoma (CCA), also known as bile duct cancer. It is an uncommon but aggressive malignancy that occurs in the epithelial cells lining the bile ducts. These bile ducts play an important role in moving bile to the small intestine from the liver, aiding in digestion. Cholangiocarcinoma accounts for approximately 3% of all gastrointestinal malignant cancers but is linked with a high mortality rate because of its late-stage diagnosis and limited treatment options.

Types of Bile Duct Cancer:

Cancer always has its origin. Thus, there are two forms of bile duct cancer depending on where the cancer is located in the bile duct system. Intrahepatic bile duct cancer occurs from bile ducts within the liver, while extrahepatic bile duct cancer occurs from outside the liver.

Intrahepatic cholangiocarcinoma (iCCA) is a rare but aggressive form of liver cancer that occurs in the intrahepatic bile ducts (a network of tubes inside the liver responsible for moving bile). While cholangiocarcinoma itself is an uncommon cancer, iCCA accounts for around 10 to 15% of all primary liver cancers and is becoming increasingly recognized worldwide.

Because of its subtle early symptoms and complex location, iCCA is often diagnosed at an advanced stage, leaving patients with limited treatment options. However, recent medicines such as Lytgobi (futibatinib) are changing the treatment landscape for a subset of iCCA patients with specific genetic mutations.

What Is Intrahepatic Cholangiocarcinoma (iCCA)?

Intrahepatic cholangiocarcinoma is a malignant tumor that begins in the bile ducts located within the liver. It is different from extrahepatic cholangiocarcinoma, which starts outside the liver. iCCA is an insidious growth that usually has no symptoms until it is quite advanced or has spread (metastasized) to distant body parts.

Risk Factors & Causes:

There are a few chronic conditions that could increase the likelihood of developing iCCA, and they are the following:

• Bile duct stones
• Chronic liver diseases (hepatitis B or C, cirrhosis)
• Primary sclerosing cholangitis (PSC)
• Parasitic infections in the biliary tract (common in Southeast Asia)
• Exposure to certain chemicals or toxins
• Genetic mutations, especially in FGFR2 (fibroblast growth factor receptor 2), are observed in a subset of iCCA cases

Signs and Symptoms of iCCA:

iCCA often occurs silently. When signs and symptoms do appear, they may include:

• Fatigue
• Weakness
• Mild jaundice (in later stages)
• Fever or night sweats
• Itchy skin
• Hepatomegaly (enlarged liver)
• Abdominal pain or discomfort
• Unexplained weight loss

These signs and symptoms may be mistaken for other liver or gallbladder conditions, which may delay early detection.

• Diagnosis of Intrahepatic Cholangiocarcinoma:
• Diagnosing iCCA involves:
• Imaging tests: MRI, CT scan, or ultrasound
• Liver function tests (LFTs) and tumor markers
• Liver biopsy for histological examination
• Molecular profiling to detect mutations like the FGFR2 gene fusion

Identifying FGFR2 fusion is important as it directly determines whether targeted therapy, such as Lytgobi may be effective.

Lytgobi® (Futibatinib): A New Therapeutic Option for FGFR2-Positive iCCA

The traditional technique for treating iCCA focused on chemotherapy utilizing gemcitabine and cisplatin, which may control the disease briefly but often leads to worsening outcomes over time.
The approval of Lytgobi® (futibatinib) for iCCA patients with FGFR2 gene fusions by the U.S. FDA represents a major milestone in iCCA care.

What Is Lytgobi®?

• Type: Oral targeted therapy (tyrosine kinase inhibitor)
• Target: Selectively inhibits FGFR2 fusion proteins that drive the growth of cancer
• Indication: Adults with advanced or metastatic iCCA with FGFR2 gene fusion, after progression on previous treatment
• Form: Oral tablets, typically 20 mg

How Lytgobi® Works?

Lytgobi® acts by strongly binding to genetically mutated FGFR2 receptors, followed by complete blockage of its functions, leading to halting the proliferation of cancerous cells, thus aiding in tumor shrinkage or stunting its growth for patients whose cancers are responsive to the mutation.

Clinical Impact of Lytgobi® in iCCA:

• In clinical investigations of the FOENIX-CCA2 trial, Lytgobi® was able to provide:
• A response rate of more than 40% to previously treated patients.
• Controllable disease progression in patients with FGFR2-positive iCCA.
• Improved progression-free survival (PFS) over conventional options

These results make Lytgobi® an important therapeutic option for patients with limited alternatives.

Accessing Lytgobi® for iCCA Patients in India and Worldwide:

Lytgobi is yet to be approved in many nations, including India. Nevertheless, it can be obtained easily through the Indian Pharma Network (IPN), as we assist in obtaining it through a Named Patient Program (NPP).

As a licensed importer, IPN facilitates access to difficult-to-obtain medicines such as Lytgobi. With our extensive knowledge of regulations, IPN’s combined network of international suppliers and cold-chain logistics guarantees safe and timely delivery of medicines to patients who qualify.

Services Include:

• Verified sourcing from approved international manufacturers
• Full CDSCO import documentation
• Support for hospitals, oncologists, and patients
• International shipping with temperature-controlled packaging
• Delivery within 7 to 21 days based on origin

Who Can Benefit from Lytgobi®?

• Lytgobi® is specifically suggested for the following patients:
• Patients suffering from FGFR2-positive intrahepatic cholangiocarcinoma.
• Individuals who have received and failed prior systemic therapy
• Those seeking personalized, molecularly guided treatment
• Hospitals and cancer centers treating rare liver malignancies

Contact Us to Access Lytgobi® for iCCA:

Intrahepatic cholangiocarcinoma (iCCA) is a relatively recent diagnosis and can be extremely debilitating for the patient, as they are often left with no suitable treatment avenues. The approval of Lytgobi® (futibatinib) is set to change the fate for many patients suffering from iCCA with an FGFR2 fusion. With targeted strategies and clinical proof, Lytgobi® is setting new benchmarks for the treatment of aggressive cancer.

For those having an advanced stage diagnosis of iCCA with an FGFR2 fusion positive result, Lytgobi® can likely be of great help. It can be accessed through Indian Pharma Network (IPN).

Disclaimer: This content is for informational purposes only. It does not replace medical advice. Always consult your treating clinician before starting any new medicine or treatment.