FDA Limits Gene Therapy Treatment Following Deaths of 2 Children | The Epoch Times

FDA Limits Gene Therapy Treatment Following Deaths of 2 Children | The Epoch Times

https://www.theepochtimes.com/health/fda-limits-gene-therapy-treatment-following-deaths-of-2-children-5945764?utm_source=truthsocial&utm_medium=Social&utm_campaign=ettruthsocial

The Food and Drug Administration approved an updated safety warning for a gene therapy treatment that restricts its use following reports of two deaths among pediatric users, the agency said in a statement on Nov. 14.

The approval was granted for Elevidys, a one-time gene therapy treatment for Duchenne muscular dystrophy (DMD) by pharmaceutical company Sarepta Therapeutics Inc. DMD renders muscle cells vulnerable to injury, leading to progressive muscle degeneration.

Elevidys is a prescription gene therapy used to treat ambulatory and non-ambulatory individuals aged at least 4 years old who suffer from DMD and have a confirmed mutation in the dystrophin gene. Ambulatory refers to individuals who can walk and are not confined to bed.

Story continues below advertisement

YOUR HEALTH MATTERS

Neuropathy is Linked to This Household Item (Stop Using It)

Nerve Recovery

OUD can be a daily struggle. Find a treatment that may help

Sponsored

The labeling update limits the “therapy’s indication to ambulatory patients,” the FDA said. The labeling must no longer contain an indication that the treatment is for non-ambulatory patients with DMD.

Furthermore, the new labeling includes a Boxed Warning—the agency’s “most prominent” safety warning.

“These actions follow reports of fatal acute liver failure in non-ambulatory patients treated with the product,” the FDA said.

Related Stories

FDA Officials Announce New Pathway for Bespoke Gene Therapies

FDA Names New Top Drug RegulatorIn a June 24 safety communication, the agency said it was investigating these incidents.

Story continues below advertisement

The two male pediatric patients who died had elevated levels of transaminase, which are enzymes released by the liver into the bloodstream. High levels of transaminases indicate that the liver is under stress.

In both cases, the patients ended up getting hospitalized two months after they received Elevidys treatment.

Following the safety communication, Sarepta voluntarily ceased the distribution of Elevidys for use in ambulatory patients, the FDA said.

In addition to the fatal cases, there has also been a non-fatal case of acute liver injury, with the individual suffering complications such as vein thrombosis, necrosis, and bowel ischemia, it said.

Story continues below advertisement

“After a comprehensive evaluation of the available safety data, FDA has now approved substantial labeling revisions for Elevidys,” the agency said.

Revised Guidelines, Company Response

Sarepta’s Boxed Warning describes the risk of “serious liver injury and acute liver failure, including fatal outcomes.”

The labeling should update the Warnings and Precautions, Adverse Reactions, Dosage and Administration, Clinical Studies, and other relevant sections, the FDA said. It must also include a new Medication Guide for patients and caregivers.

According to the FDA, the revised labeling should recommend that users conduct weekly liver function tests for at least three months after treatments, with the individuals advised to remain near a medical facility for at least two months following the therapy.

The labeling must warn patients who experience yellowing of the skin or eyes, or a change in mental status, to immediately seek medical attention. It should also contain a warning that Elevidys is not recommended for patients with preexisting liver impairments, recent or active infections, or those who have received recent vaccinations.

Story continues below advertisement

“The FDA is requiring the manufacturer to conduct a postmarketing observational study to further assess the risk of serious liver injury,” the agency said.

“The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.”

In a Nov. 14 statement, Sarepta said the company was continuing to work closely with the FDA to ensure all regulatory decisions regarding Elevidys are “grounded in science,” and in the best interests of the patients.

To date, the gene therapy has been administered to more than 1,100 individuals worldwide in both clinical and real-world settings, it said.

Story continues below advertisement

“We want to thank the FDA for their thorough and collaborative review. Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions,” said Louise Rodino-Klapac, president of research & development and technical operations at Sarepta.

In a quarterly report filed with the Securities and Exchange Commission for the period ending Sept. 30, the company said it had stopped all shipments of Elevidys in the United States effective July 22, including for ambulatory patients.

On July 28, the FDA informed Sarepta that it recommended continuing shipments of the therapy for ambulatory patients. The suspension for non-ambulatory patients remained.

WHAT IS DIRECT PAY ? CAN I USE MY HSA ACCOUNT TO PAY FOR THE MONTHLY PREMIUM

YES

DPC is a common acronym with several meanings depending on the context, including Direct Primary Care, a healthcare model where patients pay a flat fee for services;

The Direct Primary Care (DPC) model is a practice and payment model where patients/consumers pay their physician or practice directly in the form of periodic payments for a defined set of primary care services. DPC practices typically charge patients a flat monthly or annual fee, under terms of a contract, in exchange for access to a broad range of primary care and medical administrative services. The DPC practice framework includes any practice model structured around direct contracting with patients/consumers for monthly or annual fees which serve to replace the traditional system of third party insurance coverage for primary care services. Typically, these periodic payments provide patients enhanced services over traditional fee-for-service medicine. Such services may include real time access via advanced communication technology to their personal physician, extended visits, in some cases home-based medical visits, and highly personalized, coordinated, and comprehensive care administration. The AAFP supports the physician and patient choice to, respectively, provide and receive healthcare in any ethical healthcare delivery system model, including the DPC practice-setting.

The DPC contract between a patient and his/her physician provides for regular, recurring monthly revenue to practices which typically replaces traditional fee-for-service billing to third party insurance plan providers. For family physicians, this revenue model can stabilize practice finances, allowing the physician and office staff to focus on the needs of the patient and improving their health outcomes rather than coding and billing. Patients, in turn, benefit from having a DPC practice because the contract fee covers the cost of many primary care services furnished in the DPC practice. This effectively removes any additional financial barriers the patient may encounter in accessing routine care primary care, including preventative, wellness, and chronic care services. Most patients, depending on affordability, still carry insurance for coverage of healthcare services that cannot be provided in the primary care practice setting, such as specialty care and hospitalizations. The model is especially well suited for those patients with high deductible plans where they might normally be paying out of pocket for any primary care services that are not considered preventive.

Ideally, the DPC model is structured to emphasize and prioritize the intrinsic power of the relationship between a patient and his/her family physician to improve health outcomes and lower overall health care costs. The DPC contract fee structure can enable physicians to spend more time with their patients, both in face-to-face visits, and through telephonic or electronic communications mediums should they choose, since they are not bound by insurance reimbursement restrictions. For these reasons, the DPC model is consistent with the American Academy of Family Physicians' (AAFP) advocacy of the advanced primary care functions and a blended payment method of paying family medicine practices. The AAFP provides resources for members tranforming to this model, including CME credit, and will continue to promote and support Direct Primary Care as an innovative advanced practice model. (2013 COD) (September 2024 COD)

DPC practices handle lab tests by providing basic in-house tests for members and arranging external lab work at discounted rates when necessary. In many DPC clinics, blood draws are performed on-site, eliminating the need for a separate lab appointment. For specialized tests, DPC providers often have negotiated rates with national labs and may direct billing to the patient or their insurance, depending on the agreement. 

In-house testing

• Many DPC practices offer common, basic lab tests directly in the clinic.
• This allows for more comprehensive care and can include services like blood draws during a regular visit.
• Results are typically discussed in detail with the patient during the visit. 

External lab tests

• For specialized tests not performed in-house, DPC providers work with external labs.
• They often have negotiated, discounted rates for their members and may handle the billing process.
• Patients may have the option to have the test billed through their insurance, even if the DPC membership does not cover it. 

Patient access to results

• Patients can expect to receive their lab results directly, often through the DPC practice's patient portal or app.
• Many DPC systems allow patients to view their results and communicate with their doctor about them.
• In some states, consumers are also permitted to order some tests directly from a lab without a physician's order. Direct Primary Care (DPC) practices typically handle imaging by contracting with local imaging centers to provide services at a reduced cash price, often resulting in savings of 50–90% for patients. In some cases, a DPC practice may also have its own limited in-office imaging capabilities, such as an X-ray or lab services, which allows them to offer these services directly to members. These practices often leverage their cash-pay model and direct relationships with imaging providers to offer more affordable diagnostic options for their patients, especially when compared to services billed through traditional insurance. 
  How DPC handles imaging
  • Contracts with imaging centers: DPC practices can have direct contracts with local radiology and imaging centers.
  • Reduced cash prices: These contracts often allow DPC patients to pay a significantly lower cash price for services like X-rays or MRIs, with savings of 50–90% compared to traditional insurance costs.
  • In-office imaging: Some DPC practices may perform basic imaging, such as X-rays or lab work, directly in the office.
  • Direct billing: Instead of dealing with insurance companies, the patient pays the DPC practice directly, and the practice handles the payment to the imaging center.
  • Cost transparency: The DPC model makes the cost of imaging services clear and transparent, allowing patients to make informed decisions about their care. 
  Examples of DPC imaging services
  • Chest X-ray: May cost around $45.
  • MRI of the knee: May cost around $300. 
  Why this model is effective
  • Lower costs: The cash-pay model allows practices and patients to bypass the complexities and costs associated with traditional insurance billing.
  • Direct relationships: DPC practices can build strong relationships with local imaging providers, leading to better rates and more streamlined care for patients.
  • Patient-centered care: This approach allows for greater flexibility and cost savings for patients, who can focus on their health needs without the burden of traditional insurance hurdles.
  • 
     

Gene-Editing Human Embryos: A Doomed Technological Catastrophe

Gene editing is a disaster.  It goes where no man. should go.  It is the nuclear weapon of the 2000s 

Brain Fog has much to do with LIfestyle

These are the 30 truths I’ve learned about your brain:

1. Most “brain fog” isn’t age — it’s lifestyle.2. Stress is the fastest way to shrink your hippocampus.
3. Your mind is not separate from your body — they talk 24/7.
4. Sleep is the most powerful cognitive enhancer on Earth.
5. Multitasking lowers your IQ in the moment.
6. Movement is medicine for neurons.
7. Inflammation is a silent brain saboteur.
8. Emotional suppression is stored in the nervous system.
9. Your brain listens to every single thought you repeat.
10. Chronic busyness is chronic stress in disguise.
11. You can literally rewire your brain… at any age.
12. Your environment shapes your brain more than your genetics.
13. Burnout is a brain condition, not a personality flaw.
14. Focus is a skill, not a personality trait.
15. Healthy food = better neurons. Ultra-processed food = brain fog.
16. Loneliness accelerates brain aging more than smoking.
17. Consistency builds cognitive reserve — not intensity.
18. Mindfulness is neural training, not spirituality.
19. Purpose protects your brain.
20. Gut health is brain health
21. Unmanaged stress ages your brain faster than time.
22. Brain, Mind and Body need to be aligned.
23. Negativity rewires your brain to expect danger.
24. Curiosity keeps your brain biologically young.
25. Trauma rewires the brain.
26. “Motivation” is unreliable. Systems are not.
27. Boundaries protect your neurons.
28. Your daily habits matter more than your genetics.
29. Alzheimer’s is a midlife disease, symptoms just show up decades later.
30. A calm life is a high-performance brain strategy.

📌 Which one surprised you the most?

If you want weekly, science-based brain tips 👉 Join my newsletter Lifelong Brain: https://lnkd.in/dFnDQ3K3