After raising serious questions about its effectiveness, the FDA tonight approved Reata Pharmaceuticals’ drug for a rare genetic condition known as Friedreich’s Ataxia, which affects the nerves in the body, for people ages 16 and up. more.
Although not a cure, the drug known as Skyclarys (omaveloxolone), which will cost $370,000 a year, can slow the progression of this debilitating disease that begins in late childhood and affects approximately 6,000 Americans and 22,000 people worldwide. The FDA approval is based on a positive pivotal trial, showing that omaveloxolone beat placebo in terms of change in what’s called an mFARS score – a doctor-rated neurological rating scale used to measure AF disease progression.
At 48 weeks, patients treated with omaveloxolone demonstrated a statistically significant, placebo-corrected improvement of 2.40 points in mFARS in patients without pes cavus, a pre-existing musculoskeletal deformity of the foot, after 48 weeks of treatment (p = 0.014) – meeting the primary endpoint of the study. When pes cavus patients were included in the analysis, omaveloxolone produced a statistically significant, placebo-corrected mean improvement of 1.93 points in mFARS (p=0.034).
But it wasn’t all easy for Skyclarys, which is a small molecule designed to bind to a gene called Keap1 to enhance the activity of the Nrf2 protein to defuse inflammation.
In a second-quarter earnings report, Reata noted that the FDA was concerned about the efficacy of omaveloxolone, which means a decision on the key asset from the Plano, Texas-based biotech has took an additional three months to review after the company submitted additional data from its pivotal trial. .
In 2019, Reata surprised analysts with a positive reading from its pivotal omaveloxolone trial. However, that initial buzz died down a year later when the FDA said the additional data from the pivotal trial was not enough, asking the biotech to conduct an additional trial.
Reata’s $RETA stock fell about 30% after the three-month delay, and another 30% yesterday when the FDA’s top neuroscientist, Billy Dunn, left the FDA, effective immediately.
Since the approval, shares of Reata have soared more than 170% after hours trading, and Reata told investors on Tuesday that she would discuss with the FDA a possible extension of the label for patients. pediatricians under the age of 16.
It is the first approved treatment for Friedreich’s Ataxia, and several biotechs have struggled to try to treat or cure the condition. In 2016, Horizon’s $50,000-a-month drug Actimmune, already approved for two other indications, failed a Phase III trial for Friedreich’s ataxia. And Larimar Therapeutics’ Phase I program was clinically put on hold after non-human primates died during preclinical testing.