news/BIIB/2023.02.28/U.S. FDA approves Reata's rare genetic disorder drug.txt

Feb 28 (Reuters) - The U.S. Food and Drug Administration
on Tuesday approved Reata Pharmaceuticals Inc's drug
for the treatment of a rare genetic disorder that causes
progressive damage to the nervous system, sending shares up
nearly 160% after the bell.The drug, Skyclarys, is Reata's first product to gain
approval, and Jefferies analyst Maury Raycroft projected that
U.S. sales of the drug could reach $400 million by 2030.Reata estimates the disorder, called Friedreich's ataxia,
affects about 5,000 patients in the United States.Friedreich's ataxia is a neuromuscular disorder that causes
muscle weakness, loss of coordination and can lead to patients
being bound to a wheelchair in their mid-20s and premature
death.Raycroft, before the FDA decision, estimated the drug
could be priced at about $425,000 per patient annually.Reata's case for approval was based on additional data
requested by the FDA and a mid-stage study that the drugmaker
said showed patients who took the drug experienced an
improvement in neurological functions such as speaking,
swallowing and standing, compared with a placebo.The FDA said in 2020 there was not enough evidence to
support approval based on the single study, but Reata has since
then submitted additional analyses, stopping short of conducting
another clinical trial.The FDA has previously approved drugs for neurological
conditions based on limited data such as for Biogen Inc's
Alzheimer's drug Aduhelm and Amylyx's ALS
drug.Those approvals were made under the leadership of Billy
Dunn, who resigned from the agency on Monday after which Reata's
shares tumbled 30% as investors fretted about the possibility of
approval under a new division head.(Reporting by Raghav Mahobe, Mariam E Sunny and Pratik Jain in
Bengaluru; Editing by Krishna Chandra Eluri and Sherry
Jacob-Phillips)