According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the US Food and Drug Administration (FDA). This designation was given for the treatment of glioblastoma, a rare type of brain cancer, in children and adults.
Glioblastoma is a rare brain cancer. It is also the most aggressive cancer to originate in the brain. It is characterized by its rapid progression and poor response to most treatments. In most cases, the cause of glioblastoma is not known. A small number of cases evolve from another type of tumor called an astrocytoma. Risk factors for glioblastoma include genetic disorders such as Turcot syndrome and neurofibromatosis, exposure to pesticides, smoking, and a career in petroleum refining or rubber manufacture. Symptoms of glioblastoma include personality changes, headaches, memory loss, seizures, vomiting, and nausea; patients may lose consciousness in late stages. Treatment approaches include anticonvulsants, steroids, chemotherapy, radiation, and surgery. While a small number of patients can survive for several years, treatment is often ineffective, with the tumor relapsing quickly. Five year survival rate is only three percent. To learn more about glioblastoma, click here.
About Organ Drug Designation
Orphan Drug designation is generally reserved for therapies being developed to treat rare diseases, which is defined as any illness that affects less than 200,000 people in the US. To qualify, a therapy must show potential benefits in safety or efficacy in comparison to currently available treatments. Alternatively, it must have the potential to satisfy a currently unmet medical need. This designation confers several benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a period of market exclusivity lasting seven years if the drug gains FDA approval.
Trodelvy, which is currently approved to treat triple negative breast cancer, is an antibody-drug conjugate that acts on a cell-surface protein called Trop-2. This protein is expressed by many solid tumor cancers. After binding to this protein, Trodelvy delivers a drug called SN-38 in order to kill the cancer cells. This drug is being investigated for metastatic breast cancer, metastatic non-small cell lung cancer, and metastatic urothelial cancer.