Warren Huff, Reata Pharmaceuticals CEO

FDA fi­nal­ly ap­proves Reata's rare ge­net­ic dis­ease drug

Af­ter rais­ing se­ri­ous ef­fi­ca­cy ques­tions, the FDA this evening ap­proved Rea­ta Phar­ma­ceu­ti­cals’ drug for a rare ge­net­ic dis­ease known as Friedre­ich’s atax­ia, which af­fects the body’s nerves, for those 16 years old and old­er.

While not a cure, the drug known as Skyclarys (omavelox­olone), which will cost $370,000 an­nu­al­ly, may slow the pro­gres­sion of this de­bil­i­tat­ing dis­ease that be­gins in late child­hood and af­fects about 6,000 Amer­i­cans and 22,000 in­di­vid­u­als glob­al­ly. The FDA’s ap­proval is based on a pos­i­tive piv­otal tri­al, show­ing omavelox­olone beat place­bo in terms of a change in what’s known as an mFARS score — a physi­cian-as­sessed neu­ro­log­i­cal rat­ing scale used to mea­sure FA dis­ease pro­gres­sion.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.