Reata Pharmaceuticals Inc.’s newly approved treatment for Friedreich’s ataxia, Skyclarys, was given the green light by the Food and Drug Administration (FDA) on Tuesday. On Tuesday night, the company informed investors that the wholesale acquisition price of the drug will be $370,000 per year. This news sent Reata’s stock soaring, with it rising by an incredible 195% in trading on Wednesday afternoon.
Friedreich’s ataxia is a rare nervous-system disorder that affects about 4,500 diagnosed patients in the United States. The disease affects the coordination of the arms and legs and can lead to speech difficulties and hearing loss. It is a progressive disorder, meaning that symptoms worsen over time.
Reata’s Skyclarys is the first and only FDA approved treatment for the condition. The drug works by targeting a protein called frataxin, which is abnormally low in those with Friedreich’s ataxia. The drug is designed to increase the production of frataxin, which in turn helps to reduce the symptoms of the disorder.
Reata has conducted several clinical trials for Skyclarys and the results have been promising. In a phase 3 clinical trial, patients who took the drug experienced an improvement in their walking ability, as well as an increase in their energy levels. The drug was also found to be safe and well tolerated by patients.
The approval of Skyclarys is a major breakthrough for those with Friedreich’s ataxia and their families. The drug offers hope to those who are living with the condition and may help to slow down its progression. It is also a major success for Reata Pharmaceuticals, which has been working on the drug for several years.
The approval of Skyclarys is also a testament to the power of modern medicine and the advances that have been made in treating rare diseases. It is a reminder that medical research can make a real difference in the lives of people with rare diseases.
Reata Pharmaceuticals will now be able to offer Skyclarys to patients with Friedreich’s ataxia. The drug will be available at a wholesale acquisition price of $370,000 per year, making it one of the most expensive treatments available. Despite this, many patients and families will be willing to pay the price for the chance of a better quality of life.
In conclusion, Reata Pharmaceuticals’ Skyclarys has been approved by the FDA, offering hope to those with Friedreich’s ataxia. The drug is the first and only FDA approved treatment for the condition and has been shown to be safe and well tolerated by patients. The drug will be available at a wholesale acquisition price of $370,000 per year, making it one of the most expensive treatments available. Despite this, many patients and families will be willing to pay the price for the chance of a better quality of life. The approval of Skyclarys is a major breakthrough for those with Friedreich’s ataxia and their families, as well as a major success for Reata Pharmaceuticals.
