Darwin's Our Take: Regeneron’s ‘groundbreaking’ Otarmeni gains FDA approval

Regeneron’s ‘groundbreaking’ Otarmeni gains FDA approval as first gene therapy for genetic hearing loss

Tarrytown, N.Y.-based Regeneron Pharmaceuticals announced the accelerated approval of Otarmeni (lunsotogene parvec-cwha), an adeno-associated virus vector-based gene therapy and the first and only in vivo gene therapy the FDA has approved for a specific type of genetic hearing loss.

According to Regeneron, Otarmeni is the first FDA-approved gene therapy to restore a neurosensory function to normal levels. It’s also Regeneron’s first approved genetic medicine.

Otarmeni is indicated for pediatric and adult patients with severe to profound and profound hearing loss resulting from certain variants in the OTOF gene. Eligible patients must also have preserved outer hair cell function and no prior cochlear implant in the same ear.

The surgical procedure to administer Otarmeni is similar to the procedure used for cochlear implantation, according to Regeneron, and can be performed in infants.

Regeneron said OTOF-related hearing loss affects approximately 50 newborns annually in the U.S. In the past, this type of hearing loss was considered permanent and was managed with the use of devices; however, the devices do not restore the full spectrum of sound, Regeneron noted.

Dr. Dylan Chan, a pediatric otolaryngologist at the University of California, San Francisco, told The New York Times that Otarmeni “is groundbreaking,” even though OTOF-related deafness accounts for only 2% to 8% of congenital hearing loss.

“This is the first time in history that there has been a medical therapy that has enabled deaf children to hear,” he said.

The Times noted that Dr. Chan has served as a paid adviser to Regeneron and to Eli Lilly, which is also developing a gene therapy for the same type of deafness. In fact, Dr. Chan is a principal investigator in Lilly’s clinical trial of the gene therapy.  

Dr. Daniel Lee, director of pediatric otology and neurotology at the Massachusetts Eye and Ear Infirmary, echoed Dr. Chan’s viewpoint of Otarmeni as groundbreaking, according to the Times.

“We have now entered the era of biological treatment for inner ear hearing loss,” Dr. Lee said. He serves on the advisory board of Skylark Bio, a small biotech that is developing a gene therapy for a different form of genetic hearing loss.

OUR TAKE: Regeneron said it will provide Otarmeni free of charge to patients in the U.S., though CNBC reported the company hasn’t decided yet how it will price the gene therapy in other countries.

“Otarmeni … is a powerful reminder of what becomes possible when brilliant minds have the resources and freedom to pursue difficult challenges,” said Dr. George Yancopoulos, Regeneron’s cofounder, co-chair, and chief scientific officer. “Our decision to provide Otarmeni for free in the United States serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.”

Regeneron has an internal research team that’s fully committed to understanding the biology of how adults can lose hearing over time, Fierce Pharma reported.

Jonathan Whitton, Regeneron’s vice president of genetic medicines, told the news outlet: “If you think about acquired hearing loss, age-related hearing loss, or noise-induced hearing loss, these are millions and millions of people who are dealing with this. One of the challenges is people don't really understand the biology very well of exactly what's causing hearing loss.

“We’re making significant investments into deeply understanding the biology of these acquired forms of hearing loss so that we can also develop medicines in the same way we did with [Otarmeni].”

HCR #206: Embracing the Prevention Mindset with Dr. Barry R. Davis, author, Preventioneers

Health care spends trillions treating disease, but far less preventing it. The gap between what we know works and what actually gets implemented may be the most expensive problem in medicine. Barry Davis, MD Phd, Professor Emeritus of Biostatistics and Public Health, UT Houston and author of Preventioneers, joins John to explore why prevention so rarely makes it from research to reality, and what it would take for health system leaders to finally close that gap. Available on YouTube here or on all podcast platforms.

What else you need to know

Sun Pharmaceutical Industries agreed to acquire Organon in an all-cash deal with an enterprise value of $11.75 billion. Jersey City, N.J.-based Organon, which focuses on products and solutions that predominantly address women’s health, was established in 2021 through a spinoff from Merck. The company has more than 70 products in its general medicines and women’s health franchise. Sun Pharma, according to the announcement, is India’s largest biopharma firm and the world’s leader in specialty generics.

By acquiring Organon, Sun Pharma will expand its innovative medicines business and gain entry into the biosimilars market in seventh place globally, the companies said. The combined entity will have a presence in 150 countries and generate estimated annual sales of $12.4 billion, with each company contributing nearly equally to that total.

Both companies’ boards have approved the acquisition, which is subject to the usual closing conditions as well as regulatory and Organon shareholder approvals. If all conditions are met, the transaction is expected to close early next year.

Humana’s CenterWell Pharmacy business is partnering with Mark Cuban Cost Plus Drugs to develop new “end-to-end employer prescription solutions,” the companies said in a press release. Through their arrangement, CenterWell Pharmacy will become a pharmacy partner of Cost Plus Drugs and will use Cost Plus Drugs’ SwiftyRx software platform for medication order intake. The combination of the SwiftyRx technology, Cost Plus Drugs’ pass-through drug pricing, and CenterWell Pharmacy’s distribution infrastructure will lead to operational efficiencies that can result in smoother patient onboarding, automated benefit checks, and reduced costs to fill prescriptions. The companies also said the SwiftyRx platform should give CenterWell Pharmacy the ability to offer home delivery pharmacy services to eligible employees enrolled in the Humana Associate Benefit Plan.  

Cigna intends to exit the individual exchange business at the end of the year, Brian Evanko, the company’s chief operating officer, revealed during the company’s recent earnings call. Ann Dennison, Cigna’s chief financial officer, said the move is part of the company’s strategic shaping of its portfolio. Evanko said the individual exchange business is now “a small business” for Cigna and has “been shrinking in recent years.” Leaving this market will allow the company to intensify its focus on core growth platforms such as its specialty and care services, as well as its pharmacy benefit services and U.S. employer-sponsored plans. The insurer is also initiating a strategic review of alternatives for eviCore, an Evernorth subsidiary that manages medical claims and reviews prior authorization requests.  

The Department of Justice sentenced Purdue Pharma Tuesday and ordered the OxyContin manufacturer to pay more than $5 billion in criminal penalties for its role in contributing to the opioid epidemic. A federal court in Newark, N.J., ordered a criminal fine of $3.5 billion and another $2 billion in criminal forfeiture, the DOJ said in a press release. The DOJ noted that it would credit up to $1.8 billion against the $2 billion forfeiture amount based on value provided to state, local, and tribal governments through Purdue’s bankruptcy proceedings — if the drugmaker “ceases to operate in its current form and emerges from bankruptcy s a public benefit company or entity with a similar mission designed for the benefit of the American public.” The sentencing is separate from an earlier $7.5 billion settlement in connection with Purdue’s bankruptcy, which a federal court cleared in November. As part of the bankruptcy, Purdue will be dissolved and its assets transferred to a new nonprofit entity.

DC developments

The FDA launched two proof-of-concept, “real-time clinical trials” in partnership with The University of Texas MD Anderson Cancer Center, the University of Pennsylvania, AstraZeneca, Amgen, and Paradigm Health. Trial researchers will report endpoints and safety signals to the FDA in real time, as the trials progress. The goal is to accelerate the drug development timeline.

AstraZeneca is conducting one of the trials, a Phase II study of treatment-naive mantle cell lymphoma, with UT MD Anderson and UPenn. The FDA said it has received and validated signals for this trial through Paradigm Health, establishing the feasibility of the technical framework needed for real-time signal sharing. Amgen will conduct the other trial, a Phase Ib study of limited-stage small cell lung carcinoma. The agency also released a request for information on a proposed pilot program of real-time clinical trials.

CMS’ pilot program to test the use of AI-driven prior authorization (PA) in Medicare is delaying care and creating burdens for patients and providers, according to a letter Sen. Maria Cantwell, D-Wash., sent Health Sec. Robert Kennedy, Jr. The Wasteful and Inappropriate Service Reduction (WISeR) model launched at the start of the year and is scheduled to run for six years. In the six states participating in the program, patients enrolled in traditional Medicare are subject to PA requirements for 13 types of medical services. CMS contracts with private companies to manage the PA requests.

Sen. Cantwell’s office released a report with new data from the Washington State Hospital Association indicating that patients are waiting two to four times longer to undergo procedures because of the delayed PA responses. Although CMS committed to responding within 72 hours, providers have reported waiting an average of 15 to 20 days for responses.

Further, the report states, hospitals throughout Washington have reported that denials are often inconsistent with clinical criteria and lack clear rationales, increasing the number of appeals submitted. Hospitals have added staff and increased hours dedicated to PA processes, raising the cost of care. Sen. Cantwell also wrote in the letter that the WISeR model financially rewards contractors for delaying or denying care.

A proposed bill to reform Medicare Advantage has bipartisan, bicameral support in Congress. Earlier this month, the Medicare Advantage Improvement Act was introduced in the House of Representatives by seven lawmakers, and last week, Sens. Roger Marshall, R-Kan., and Sheldon Whitehouse, D-R.I., introduced the legislation in the Senate. Of note, five of the seven representatives, as well as Sen. Marshall, are physicians.

The bill would address issues that currently plague MA enrollees, such as delayed approvals, surprise denials, confusing appeals processes, and disruptions to care. Provisions in the bill call for steps such as tightening timelines for prior authorization responses, requiring real-time authorization decisions for some services, imposing stricter denial policies, and establishing a compliance scoring system for MA plans that could result in automatic payment cuts for low scores.

What we’re reading

Artificial Intelligence Is Not the End of the Physician. JAMA, 4.29.26 (subscription or registration required)

AI Won’t Fix Your Health System. Redesigning It Will. BCG, 4.26.24

How to Transform a Health System’s Organizational Culture and Hardwire It for the Future. NEJM Catalyst, 4.15.26 (abstract available; subscription required for full article)