The FDA has chosen seven companies, including Eli Lilly and Regeneron, for its PreCheck pilot program to speed up reviews of new domestic pharmaceutical manufacturing facilities. The initiative aims to cut review times by up to 14 months by allowing construction-phase inspections.
Regeneron will provide its newly approved gene therapy Otarmeni for free in the US, treating a rare genetic hearing loss caused by faulty OTOF gene. In clinical trials, 80% of patients experienced hearing improvements. CEO Leonard Schleifer said other countries have not yet been priced but should contribute their fair share. About 50 babies annually are born with this condition in the US.
The FDA approved the first gene therapy to restore hearing for people born deaf, developed by Regeneron Pharmaceuticals. The treatment targets a rare genetic defect affecting about 50 U.S. children annually, caused by a defective OTOF gene. In clinical trials, 80% of patients achieved significant hearing restoration and 42% gained normal hearing, with effects lasting at least two years.
Regeneron agreed to lower U.S. drug prices for some Americans under a deal with President Trump, part of his 'most favored nation' effort to align U.S. prices with lower international rates. The biotech company will also offer its newly FDA-approved hearing-loss gene therapy Otarmeni for free to eligible patients. The agreements exempt companies from tariffs for three years, including planned up to 100% levies on pharmaceuticals. The FDA approved Otarmeni under its National Priority Voucher program for an ultra-rare genetic condition causing deafness.
