Because there are many obstacles that stand in the way of developing drugs for rare diseases, the FDA grants a number of designations to incentivize companies to create these treatments. Recently, the regulatory agency has granted its Rare Pediatric Disease designation to IMX-11o, a therapy for rhabdomyosarcoma.
First off, it’s important to understand just what this rare disease is. Rhabdomyosarcoma is a rare form of childhood cancer that begins in the soft tissue. While it most commonly affects children, people of any age may be impacted. It leads to varying symptoms depending on where in the soft tissue the tumor forms, but possible symptoms include blood in the urine, issues with bowel movements, trouble urinating, swelling in the eyes, headaches, bleeding in the throat, nose, and ears; and masses or bleeding in the vagina or rectum.
Medical professionals do not know what causes this cancer, although they have identified a number of risk factors, such as a family history of cancer, Noonan’s syndrome, and Costello syndrome. In terms of treatment, a combination of therapies is typically required. Options include radiation therapy, chemotherapy, surgery, and clinical trials.
IMX-110 for Rhabdomyosarcoma
Now that we have an understanding of rhabdomyosarcoma, let’s move on to IMX-110. This therapy combines a poly-kinase inhibitor and apoptosis inducer, and it is able to deliver these components deep into a tumor’s microenvironment. According to its manufacturer, ImmixBio, the drug’s goal is to cut the tumor off from both its structural and metabolic support, effectively severing its lifeline.
Upon seeing data on IMX-110 and the severe unmet need faced by rhabdomyosarcoma patients, the FDA granted it the Rare Pediatric Disease designation. This designation is reserved for therapies that treat serious conditions impacting less than 200,000 people in the United States and which mainly affect children. When it is given to a company, it also comes with a priority review voucher upon approval of a new drug application (NDA).
Hopefully, the development of IMX-110 continues to be positive, as it could fill an unmet need faced by rhabdomyosarcoma patients across the country.
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