Reata’s Rare Genetic Disorder Drug gets US FDA Approval

HEALTHCARE

Reata Pharmaceuticals Inc.’s drug for the treatment of a rare genetic disorder that causes progressive damage to the nervous system was approved by the U.S. Food and Drug Administration (FDA), prompting its shares to rise nearly 160% after the opening bell.

Skyclarys, Reata’s first approved product, is expected to sell for $400 million in the United States by 2030, according to Maury Raycroft, a Jefferies analyst.

According to Reata’s estimation, around 5,000 people in the United States suffer from the condition known as Friedreich’s ataxia.

A neuromuscular disorder known as Friedreich’s ataxia can result in muscle weakness, loss of coordination, and a wheelchair-bound patient in their mid-20s or even a premature death.

Prior to the FDA’s decision, Raycroft estimated that the drug could cost $425,000 per patient annually.

The FDA requested additional data, and a mid-stage study showed that patients who took Reata had better neurological functions like speaking, swallowing, and standing than those who took a placebo, according to the company’s case for approval.

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In 2020, the FDA stated that there was insufficient evidence to warrant approval based on the single study; however, Reata has since submitted additional analyses without initiating a second clinical trial.

Aduhelm, a drug developed by Biogen Inc. for the treatment of Alzheimer’s disease, and Amylyx’s ALS, a drug developed by Amylyx, have both received prior FDA approval on the basis of sufficient data.