An Experimental Treatment for Adenoid Cystic Carcinoma Earns Orphan Drug Designation

According to a story from, the drug developer Ayala Pharmaceuticals has recently announced that the company’s investigational product candidate, known as as AL101, has earned Orphan Drug Designation. This designation, issued by the US Food and Drug Administration (FDA), is for the treatment of the rare cancer adenoid cystic carcinoma. Ayala is focused on the development of therapies for genetically linked cancers.

About Adenoid Cystic Carcinoma

Adenoid cystic carcinoma is a rare type of cancer that usually appears in the salivary glands. However, it can appear in a number of different areas, such as trachea, lungs, and brain. This cancer has a genetic basis and is triggered by the activation of the MYB gene, which codes for an oncogenic transcription factor. Adenoid cystic carcinoma is not inherited and this activation is usually the result of external factors. This is a slow growing cancer and patients often survive for several years, even if the cancer has metastasized. Symptoms vary depending on the location, where a painless lump or mass is present. Other symptoms include pain or nerve paralysis. The best treatment for this cancer is surgical removal of the tumor, often supplemented with radiotherapy. When surgery is not an option, fast neutron therapy is regarded as the best option; chemotherapy may be used when the disease begins to spread. To learn more about adenoid cystic carcinoma, click here.

About Orphan Drug Designation

Orphan Drug Designation is reserved for treatments that are in development for rare diseases, which is legally defined as any disease that affects less than 200,000 people in the US. To qualify, a drug must display the potential to fulfill a currently unmet medical need or have significant advantages in effectiveness over currently available therapies. This designation confers several benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a period of market exclusivity to last seven years if the drug gains approval from the FDA.

AL101 is classified as a gamma secretase inhibitor. Gamma secretase is a type of Notch signaling pathway, which plays a critical role in tumor development for a number of different cancer types. The drug is currently being tested in a Phase 2 trial as a treatment for adenoid cystic carcinoma.

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