The United States Food and Drug Administration has awarded Orphan Drug Designation to Q-Cells®, a product being developed by Q Therapeutics, for the treatment of transverse myelitis. For more detailed information you can read the source press release at Globe Newswire by clicking here.
About Transverse Myelitis
Transverse myelitis is an inflammation of the spinal cord. According to the NIH, it can be caused by factors such as infections, immune system disorders, and other conditions that damage myelin (the substance that insulates nerve fibres). Spinal cord inflammation can prevent nerves from sending messages properly, which may impact a person’s nerve signalling below the inflammation. People with transverse myelitis may experience pain, weakness, and sensory problems, which can develop extremely suddenly (over several hours), or less suddenly. Some people recover from transverse myelitis, while others experience lasting impairments.
Q Therapeutics’ investigational Q-Cells product has been researched using mouse models, which showed that the Q-Cells were able to replace the missing myelin and restore the mice to normal function and life expectancy. This encouraging data supported FDA clearance of a planned clinical trial.
Orphan Drug Designation
Q-Cells have now received Orphan Drug Designation from the FDA for the treatment of transverse myelitis. Orphan Drug Designation is granted to drugs that are being developed to treat rare conditions, which is typically considered to be a condition or subset of a condition that affects 200,000 or fewer people in the US.
Being awarded Orphan Drug status gives the developers of the drug or product several benefits, which are designed to speed up the process of bringing the drug to patients. These benefits may include tax credits for clinical testing, waived prescription drug user fees, and, if the drug is approved by the FDA, a seven-year period of marketing exclusivity. For more information about Orphan Drug status, click here.