Regeneron $REGN said that continued approval may depend on confirming the clinical benefit in the next part of the CHORD trial. The most common side effects were middle ear infection, vomiting, nausea, dizziness, pain from the procedure, trouble walking, and rapid, involuntary eye movements.
Regeneron developed Otarmeni with Decibel Therapeutics, a smaller biotech it acquired in 2023 for about $100 million, according to Bloomberg. Across the gene therapy sector, single-administration treatments for rare diseases have routinely been priced between $2 million and $3 million, according to Bloomberg.
"We believe that we are a very different type of company," Regeneron President and CSO George D. Yancopoulos said in a statement. According to CNN, Yancopoulos disclosed that a $4 million-per-child price point had been debated internally — a figure that, across the roughly 50 annual U.S. cases, would have translated to between $200 million and $400 million in yearly revenue, according to CNN.
Regeneron noted that while the therapy itself will be free, out-of-pocket costs for the surgical administration procedure remain outside the company's control. Patients can contact the company's OnPath patient support program for access information.
Timing the announcement alongside the FDA clearance, Regeneron also formalized a drug-pricing deal with the Trump administration — a move that brought the company in line with a wave of pharmaceutical firms that had previously agreed to narrow the gap between U.S. and international drug prices, according to Bloomberg. Under the terms of the deal, the company committed to cutting what it charges Medicaid for certain medicines and offering its cholesterol treatment, Praluent, at a reduced price through the TrumpRx program.
Otarmeni received several special designations from the FDA, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy. It was also one of the first medicines chosen for the FDA Commissioner's National Priority Voucher program, which speeds up reviews for drugs that address important health needs. Regeneron said it plans to seek approval in more countries.
