
Children's Hospital LA pauses use of Sarepta gene therapy for all patients
The FDA on Friday asked Sarepta to voluntarily halt shipments of the gene therapy after a muscular dystrophy patient who received a different, experimental treatment died. The company said it did not plan to comply with the request and would continue making the treatment available to ambulatory patients.
The company in June suspended use of Elevidys for patients whose disease had progressed to the point where they need a wheelchair after two teenagers died from liver failure after treatment.
Elevidys has full FDA approval for children with Duchenne Muscular Dystrophy who are still able to walk, but was given conditional approval for non-ambulatory patients.
Children's Hospital LA said its pause, effective July 18, has been communicated to "affected patient families while it awaits any further determination by the FDA."
The hospital is one of more than 70 authorized to administer Elevidys. Five other major children's hospitals asked by Reuters to comment on whether they planned to continue use of the gene therapy did not immediately respond.
Shares of Sarepta, down about 90% year-to-date, fell 5% on Monday to close at $13.32 on Nasdaq.
Hashtags

Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles


Reuters
14 minutes ago
- Reuters
HCA lifts 2025 profit forecast, flags insurance policy uncertainty
July 25 (Reuters) - HCA Healthcare (HCA.N), opens new tab lifted its 2025 profit forecast but said it cannot determine how looming changes to insurance plans under Medicaid and Obamacare will impact 2026 earnings, sending the hospital operator's shares down 2% on Friday. Some COVID-era subsidies under Obamacare are set to lapse in 2026. That would impact patient coverage, lead to a spike in insurance premiums and cause a drop in enrollment numbers, leaving hospital operators such as HCA to foot a heftier bill for compensated care. "We continue to advocate strongly for their extension, but at this point we do not know what the outcome will be," CEO Samuel Hazen said during a call with analysts. Hazen said the company is working on a cost efficiency initiative to offset any impact from the subsidy expiry and other government actions such as Medicaid policy changes and tariffs. Still, the hospital chain operator raised its 2025 profit forecast to between $25.50 and $27 per share, from a range of $24.05 to $25.85 earlier. The latest forecast includes the expected impact from the Trump administration's current and future policies, including potential tariffs on imports. It also posted upbeat second-quarter results. Quarterly revenue rose 6.4% to $18.61 billion, compared with analysts' estimates of $18.50 billion, according to data compiled by LSEG. Adjusted profit came in at $6.84 per share, beating estimates of $6.25. However, HCA's same-facility inpatient and outpatient surgeries decreased by 0.3% and 0.6%, respectively, in the quarter ended June 30. Investors appeared concerned about upcoming regulatory changes that could lead to lower medical membership on the Obamacare and Medicaid plans, said Morningstar analyst Julie Utterback. "Share reaction may reflect that profit growth could slow materially for caregivers like HCA in the next couple of years." Utterback said.


Reuters
44 minutes ago
- Reuters
US FDA extends review of Bayer's menopause relief drug
July 25 (Reuters) - The U.S. Food and Drug Administration has extended its review of Bayer's ( opens new tab experimental menopause relief drug, the German drugmaker said on Friday. The non-hormonal treatment, elinzanetant, is being reviewed for relieving moderate to severe vasomotor symptoms, also known as hot flashes, associated with menopause. The FDA has extended the review by up to 90 days and did not raise any concern regarding the general approvability of the drug, Bayer said. The drug, branded as Lynkuet, is approved in the United Kingdom and Canada.


Reuters
an hour ago
- Reuters
US FDA says J&J's unit issues correction related to surgical stapler
July 25 (Reuters) - The U.S. drug regulator said on Friday a unit of Johnson & Johnson (JNJ.N), opens new tab issued a correction for certain lots of a part related to its surgical stapler and classified the action as "most serious". The issue is related to the unit Ethicon Endo-Surgery's device, 'Endopath Echelon Vascular White Reload for Advanced Placement Tip', which tends to inadvertently lockout during surgical procedures, the Food and Drug Administration said, opens new tab on its website. "There has been one reported death and one injury related to this issue," the agency added. The part in question is a single-use cartridge that surgeons load into a stapler to cut and close blood vessels or tissue during an operation. This helps control bleeding and close wounds quickly. However, the FDA said the device may sometimes appear to work but fail to cut or staple tissue during surgery, and could lead to life-threatening bleeding, surgical delays, or death. A J&J spokesperson said in an email to Reuters that it notified its affected customers through a correction letter in April, and added that it was a voluntary correction, not a product removal. Ethicon has advised healthcare facilities to review affected batches and ensure operating room personnel are familiar with the device's instructions for use and lockout mitigation protocols, the FDA said.