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KeYe Life's KY1 molecule has received orphan drug designation from the FDA for hepatocellular carcinoma
Source: | Author:开悦生命 | Published time: 2024-10-22 | 455 Views | Share:

On June 25, 2024, Keye Life's KY1 drug received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for hepatocellular carcinoma (HCC). The application number is DRU-2024-10075. The mechanism of KY1 is different from existing drugs for HCC, as it targets the RNA helicase DHX33 protein. An analysis of 520 liver cancer patients revealed that DHX33 protein is highly expressed in cancerous tissues while being negative in adjacent normal tissues. The expression of DHX33 protein is closely related to tumor size and alpha-fetoprotein (AFP) levels. Kaplan-Meier survival curves indicated that patients with high DHX33 expression had shorter survival times and higher recurrence rates.


Although the total number of HCC patients in the U.S. is less than 200,000, China remains one of the countries with a high incidence of HCC. Currently, targeted drugs approved for the treatment of advanced HCC include first-line therapies (such as sorafenib, lenvatinib, and atezolizumab combined with bevacizumab) and second-line therapies (such as regorafenib, cabozantinib, ramucirumab, nivolumab, and pembrolizumab). Sorafenib was the first systemic oral medication targeting multiple kinase sites approved by the FDA in 2005 and remains a first-line treatment option. Lenvatinib (another multi-kinase inhibitor) was approved by the FDA in 2018 for patients with some degree of sorafenib resistance. Additionally, atezolizumab was the first immunotherapy approved for use in combination with bevacizumab for the treatment of unresectable or metastatic adult HCC. Second-line drugs such as regorafenib and cabozantinib are used for patients who have previously received sorafenib treatment and are resistant to it. Despite the variety of first-line and second-line treatment options, resistance remains the main reason for treatment failure during targeted therapy for HCC.


The FDA has five special designations for drug development: orphan drug, fast track, accelerated approval, priority review, and breakthrough therapy. The Orphan Drug Act was introduced in the U.S. in 1983 to encourage the development of drugs for rare diseases. Its incentives include tax credits (25% of clinical research costs), grants for orphan drug development projects, waivers of NDA/BLA application fees (up to over $3 million), and an extended market exclusivity for a  period of seven years. Orphan drug applicants can collaborate more closely with the FDA and are allowed to conduct disease-specific clinical trial designs to shorten the time between IND and NDA approval. The FDA even encourages the use of innovative clinical trial methods. Studies have shown that orphan drugs have a higher probability of success in clinical trials and FDA approval compared to non-orphan drugs.


The orphan drug designation for the KY1 molecule is significant for the later development of the drug, as the economic incentives associated with orphan drugs, combined with their higher clinical trial success rates, can better facilitate the company's drug development process.