Orforglipron

The FDA on Thursday granted accelerated approval to Regeneron's hearing loss gene therapy Otarmeni (lunsotogene parvec-cwha), about six months after the treatment was awarded a Commissioner's National Priority Voucher (CNPV). It's the first gene therapy to win approval under the agency's speedy new review programme and the second new molecular entity, behind the clearance of Eli Lilly's oral weight-loss drug Foundayo (orforglipron) earlier this month.Otarmeni, formerly known as DB-OTO, is an AAV vector-based gene therapy designed to deliver a full-length copy of the OTOF gene — necessary for the inner ear's sensory cells to communicate with the auditory nerve — to hair cells in the inner ear. The treatment is infused into the cochlea in a similar procedure to that used for cochlear implantation. The gene therapy was approved specifically to treat children and adults with OTOF-related severe-to-profound sensorineural hearing loss at any frequency above 90 decibel hearing level (dB HL). Patients must have preserved outer hair cell function and no prior cochlear implant in the same ear; Otarmeni is not recommended for those with an inaccessible inner ear.Regeneron said it will provide Otarmeni to US patients for free. The gene therapy's approval came just hours before the biopharma finalised its most favoured nation drug-pricing deal with the Trump administration, the seventeenth and final drugmaker that received a letter last year from the US president to strike an agreement. More than a whisper of efficacy The accelerated approval was based on the results of the Phase I/II CHORD study. The pivotal trial enrolled 20 individuals between 10 months and 16 years of age to receive an intracochlear infusion of Otarmeni either in one ear, or both. After 24 weeks, 16 (80%) of the patients experienced hearing improvements per pure tone audiometry assessments at a threshold of ≤70 dB HL — the clinical standard for natural hearing where a cochlear implant is not required — meeting the primary endpoint. Otarmeni also succeeded on a key secondary measure. Using electrical brainstem signals to measure the response to sound, 14 (70%) of participants achieved an auditory brainstem response (ABR) at ≤90 decibels at 24 weeks. Of the 12 study participants with 48-week follow-up data, five (42%) were able to hear whispers at or below 25 dB HL. "Reinstating 24/7 natural hearing is now possible," said A. Eliot Shearer, an otolaryngologist at Boston Children’s Hospital and an investigator on CHORD. "I’ve witnessed firsthand my trial participant responding to their mother’s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss."According to Regeneron, OTOF-related hearing loss occurs in about 50 newborns per year in the US.

Pictured: Sign of U.S. Department of Health & Huma iStock, mohd izzuan Two of the biggest insurance providers have expressed reluctance to participate in the government’s BALANCE program that would have made GLP-1 drugs more affordable to patients. The Centers for Medicare and Medicaid Services has indefinitely postponed the pilot run of its new BALANCE financing model that would have improved patient access to weight-loss medicines. The delay, analysts say, could have a limited but nevertheless negative impact on the bottomlines of biopharma’s obesity frontrunners. Eli Lilly ended Tuesday’s trading session nearly 2% lower than its previous closing price, while Novo Nordisk took a 4% hit. The decision to postpone, which the agency announced in an email that has since been made public on LinkedIn , comes after CVS and UnitedHealth Group, two of the biggest insurance providers in the U.S., expressed reluctance to join BALANCE, according to reporting from Bloomberg News . CVS has already declined to participate, according to the publication, while Bobby Hunter, chief of government programs at UnitedHealth, told investors during the company’s earnings call on Tuesday that “there are some notable challenges and outstanding questions with the currently planned structure.” “We believe today’s announcement where CVS has declined to participate in CMS’ BALANCE program could have a limited, negative effect of up to ~$500M in peak incretin sales for Zepbound/Foundayo Medicare Part D sales,” Truist Securities told investors in an April 21 note. If all pharmacy benefit managers opt out of BALANCE, however, the revenue hit on Lilly could balloon to around $3.3 billion, the analysts said. Still, this “potential future revenue impact would be offset if future Medicare Part D patients opted instead to purchase Zepbound/Foundayo through LillyDirect or comparable telehealth channels,” Truist added. GLP-1 Lilly’s Foundayo reaches 1,390 patients in first week, trailing Novo’s oral Wegovy launch While Novo Nordisk’s Wegovy pill reached more than 3,000 patients in its first week on the market, analysts at RBC Capital Markets said a direct comparison of the two figures could be misleading given the shorter data collection time for Foundayo. April 17, 2026 · 1 min read · Tristan Manalac Read more BMO Capital Markets likewise had a balanced view of the news. UnitedHealth’s comments on the call, according to the analysts, “are more reflective of a negotiating posture” rather than an outright refusal to participate. “Nothing in [UnitedHealth’s] response suggested a retreat from GLP-1 coverage,” the firm said. “Against this backdrop, we think the move in Lilly and Novo shares could be overdone,” BMO added in its note. BALANCE is the CMS’ proposed payment model for GLP-1 drugs, under which the agency would negotiate directly with manufacturers on behalf of state Medicaid offices and Part D plans. The goal is to make these drugs more affordable and accessible to patients. State agencies had until January 1, 2027, to signify their intent to participate in BALANCE, after which the program would kick in. CMS had planned an interim measure called Bridge, running from July 1, 2026, through the end of the year that would provide eligible Part D beneficiaries access to certain GLP-1 drugs before BALANCE goes live. With the delay on Tuesday, Bridge has now been extended to December 31, 2027, according to the CMS’ email.