Reata’s Skyclarys is a treatment for Friedreich’s ataxia, a rare inherited disease that causes damage to the nervous system
The U.S. Food and Drug Administration on Tuesday approved Reata Pharmaceuticals Inc.’s treatment for Friedreich’s ataxia, a rare inherited disease that causes damage to the nervous system.
Reata’s RETA stock was halted in after-hours trading on Tuesday in advance of the FDA’s decision.
The drug, omaveloxolone, which is now called Skyclarys,…
The U.S. Food and Drug Administration (FDA) has recently approved Reata Pharmaceuticals’ experimental drug omaveloxolone, to treat an inherited form of primary mitochondrial disease. The approval came amid questions surrounding theongoing changes to the regulatory environment for neuroscience treatments.
Primary mitochondrial disease is a rare and often fatal disorder in which the person affected cannot break down food or convert energy. In clinical trials, the drug proved effective in significantly improving the energy production and survival rate.
This is the first drug to be approved by the FDA specifically for this type of disease. The approval is also seen as a reflection of the ongoing changes in the regulatory environment for neuroscience treatments. In the past, new drugs have been subjected to lengthy and costly clinical trials, but the FDA has recently allowed for new drugs to be approved on the basis of smaller studies and, in some cases, real-world data.
The approval of the drug is expected to provide a big boost to Reata, as well as to companies developing other treatments for rare diseases. The FDA’s move is seen as an important step in the development of treatments for these diseases, which are often neglected by the pharmaceutical industry due to the small size of their patient populations.
Despite the approval, some experts have raised concerns about the drug’s safety, particularly given that the clinical trials were relatively small. Some have also raised concerns about the impact of the new regulatory environment on patient safety.
However, the FDA has defended its decision to approve the drug, noting that the data collected were sufficient to demonstrate the drug’s efficacy and safety. The FDA is also urging drug companies to continue to conduct clinical trials to further validate the safety and efficacy of the drug.
Overall, the approval of omaveloxolone marks an important milestone in the development of treatments for rare diseases and reflects the recent changes in the regulatory environment for neuroscience treatments. This approval could open up many opportunities for similar treatments for rare diseases, providing hope to many patients and their families.