Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.
Sarepta’s Elevidys will remain on the market with a black box warning. But this gene therapy for Duchenne muscular dystrophy still faces commercial headwinds and analysts say the company has work to do to win over patients and investors.
At a time when AI is reshaping pharma, Reverba Global CEO Cheryl Lubbert explained in an interview why empathy, context, and ethics still require a human touch.
The FDA approved Bayer’s Kerendia for reducing the risk of heart failure in patients with mildly reduced ejection fraction, a measure of how much blood the organ can pump. The daily pill was first approved in 2021 for patients with chronic kidney disease associated with type 2 diabetes.
The FDA published more than 200 complete response letters, the first step in what it describes as part of its effort to modernize the agency and improve transparency. But there are some caveats to the release of the FDA correspondence.
Capricor Therapeutics said the FDA asked for more data supporting the efficacy of Deramiocel, the biotech’s off-the-shelf cell therapy for treating the heart complications of Duchenne muscular dystrophy. Those data could come from an ongoing Phase 3 study expected to yield preliminary results soon.
AI is no panacea, but it can have a role in turning artisanal, trial-and-error drug development into a rapid, cost-effective, data-driven process.
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KalVista Pharmaceuticals’ Ekterly is now FDA approved for treating acute swelling attacks from hereditary angioedema. The KalVista tablet provides an alternative to injectable or infused HAE medications from companies such as Takeda Pharmaceutical and CSL Behring.
Dizal Pharmaceutical’s Zegfrovy is now FDA approved for treating non-small cell lung cancer driven by a particular rare genetic signature. Johnson & Johnson drug Rybrevant already addresses this genetic signature as an earlier line of therapy, but Dizal executives contend their drug offers the potential for better safety and efficacy.
Bispecific antibodies are already available for treating multiple myeloma. FDA approval of Regeneron Pharmaceuticals’ new drug, Lynozyfic, comes with dosing flexibility that sets it apart from others in its class.
The FDA has eliminated a safety monitoring requirement and eased certain restrictions that had been in place since CAR T-therapies first reached the market in 2017. The agency and financial analysts say the changes should improve patient access to these personalized cancer treatments.
The home care industry is booming. As Baby Boomers age and more families seek in-home solutions for loved ones, demand for home care services is at an all-time high.
Gilead Sciences drug lenacapavir, brand name Yeztugo, is now approved for HIV pre-exposure prophylaxis. The twice injectable medication is hoped to improve uptake and adherence to PrEP.
To be eligible for the FDA’s new Commissioner’s National Priority Voucher program, a drug must meet certain national health interests. Qualifying medicines could be reviewed in one to two months, down from the standard 10 months to a year.
Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys. The company has proposed an immunosuppressive regimen to mitigate the liver injury risk posed by the therapy.
FDA approval of Merck’s RSV antibody drug, Enflonsia, makes it a direct competitor to Sanofi’s Beyfortus. The new Merck product is expected to be discussed by the CDC’s Advisory Committee on Immunization Practices; ACIP will have an entirely new roster at its next meeting after all 17 sitting members were dismissed by Health and Human Services Secretary Robert F. Kennedy Jr.
May has seen a slew of executive hires, exits and layoffs across the healthcare industry. For example, the American Medical Association and TigerConnect named new CEOs, and Google and UnitedHealth Group have seen key executives leave their roles. There were also layoffs at organizations including Devoted Health and New York-Presbyterian.