FGFR, fibroblast growth factor receptor (FGFR), is one of the most popular targets for "unlimited cancer" therapy. It is an important member of the receptor family of tyrosine kinases, which consists of four highly conserved transmembrane receptor tyrosine kinases (FGFR1-4) and a receptor FGFR5 (also known as FGFRL1) that binds to FGF ligands but lacks an intracellular kinase domain.
Normal physiological conditions, FGFR and its ligand fibroblast growth factor (FGF), FGFR dimers was synthesized and its phosphorylation, activation of downstream signaling pathways, such as the JAK/STAT pathway, phospholipase C, these signal transduction pathways in multiple physiological processes such as cell proliferation, differentiation, migration and apoptosis in both plays a vital role.
However, FGFR is susceptible to various somatic aberrations, and common mutations such as overexpression, point mutation, and gene translocation occur, leading to carcinogenesis. Studies have found that FGFR gene variants are generally widespread in solid tumors such as lung cancer, liver cancer, intrahepatic cholangiocarcinoma, breast cancer, gastric cancer, uterine cancer and bladder cancer, and the types and frequencies of FGFR mutations are also different among different cancer types.
4 models have been approved, the potential market to attract continuous cooperation
FGFR inhibitors can inhibit tumor growth by blocking FGFR-mediated signaling pathway. In recent years, FGFR inhibitors have entered the harvest period, and one inhibitor has been approved every year since 2019. Up to now, 4 inhibitors have been approved globally, of which only Pemazyre has been approved in China.
The four FGFR inhibitors that have been approved are administered orally once daily, primarily for cholangiocarcinoma. None of the four FGFR inhibitors has entered the blockbuster category. However, Pemazyrehad sales of $25.9 million in 2020 and $68.5 million in 2021.
And the industry very bullish on FGFR inhibitors, NatureReviewDrugDiscovery Balversa2024 is expected annual sales is expected to reach $1.2 billion, EVP predict Truseltiq2026 annual sales of up to $253 million.
However, FGFR is the next "unlimited cancer" new target, related inhibitors are self-evidently expensive.
Balversa is priced between $10,080 and $22,680 per treatment course for 28 days. Pemazyre is listed at $17,000 per course of treatment, with an average treatment duration of about six months and eight or nine sessions, costing about $136,000 to $153,000 per patient. Truseltiq costs $21,500 a month, or about $129,000 every six months. The high cost of treatment makes ordinary patients "prohibitive", can only see from a distance.
However, as a potential stock, and the industry is optimistic about the market prospects; Major pharmaceutical companies have entered the market to cooperate in the development of FGFR inhibitors. For example, Balversa was discovered by Astex, and in 2008 Johnson & Johnson reached an exclusive global licensing and cooperation agreement with Astex for the drug.
Pemazyre was developed by Incyte. In 2018, Cinda Biotics reached a cooperation with Incyte to obtain the clinical development and commercialization authorization for three single or combined drugs including Pemazyre in Mainland China, Hong Kong, Macau and Taiwan.
Truseltiq was introduced by BridgeBio from Novartis Novartis in 2018, and BridgeBio itself and "Helsinn" will jointly be responsible for the drug's commercialization in the U.S. market and "exclusive" commercialization of the drug itself outside the U.S. (excluding Greater China). In 2020, Lentobio entered into a strategic alliance with BridgeBio to be responsible for the clinical development, registration application and future commercial operation of the drug in Greater China (Mainland China, Hong Kong, Macau), the development and commercialization of the Chinese market.