Safety – a word we hear everyday , every second  in our life .

The real question is that we use this word so frequently and even without understanding its real impact/implication or with any major understanding and that too in a variety of languages.

Right from the time human life descends on the earth, “ the Safety” mechanism starts and continues till the child moves into its destined home. At home , the concept of safety converts onto various “shapes “- be it related to culture , motherhood, parenthood, environment, upbringing, healthcare, etc.

The young life which has taken its roots on earth does not even know till he/she achieves an age of understanding life.

By the time things roll out – as the young life emerges as a “healthy” individual- he/ she start devising their own concepts to which “safety “ is no exception. This may or may not be in alignment with the concepts of growing up – yet they take some defined it undefined form.

While buying commodities to buying white goods to travelling to eating to exercising and right upto the bed and the pillow on which we take our rest, the word “safety “ never leaves us . Safety is like our second “skin “ – a cover modulated by environmental factors, people, word if mouth and what not …

This is all fine till we buy things we think we can hypothetically “control”- … as we have taken this from a renowned place/brand / seen online reviews, etc.  But who actually “validates “ this “ safety “ concept ….

We do not wait for that to happen as the moment something goes “malfunctioning “related to our purchases, we start replacing the commodity/ find solutions by switching to other options. Alternatively, we also start “condemning “ the products and the people who sold it with a hope of self-satisfaction. We forget all durability and “safety “ jargons which were told to us during the purchase.

I would like to draw your attention to “Safety” in healthcare is no exception to the above rule, particularly during medicine consumption.

As human beings our “safety “concepts are at the peak when it comes to an illness, especially a sudden one.

Medicines on the other hand, should be consumed or prescribed with extreme precautions especially the age is either too young or too old.

Patient awareness in terms of safety particularly in India, is still an emerging concept.

Multinational pharmaceutical companies publish medicine consumption and a safety information data on product specific websites. Patient safety information is also circulated by respective government agencies.

Several websites offer valuable patient information related to the pharmaceutical industry, including resources for drug information, clinical trial details, and patient support programs. Key websites include Drugs.com for drug information, MedlinePlus for general health information, and various pharmaceutical company sites for specific product details and support programs.

But when it comes to “Patient safety “ in clinical trials, a “LOT” more than safety needs to be undertaken by the companies and the institutions which are involved in clinical trial conduct.

A concise summary of the major clinical trial failures from 2024

Summary: 5 Major Drug Development Failures in 2024

Key Insights:

• Late-stage failures were mostly due to lack of efficacy or safety issues.
• Neurology and oncology remained high-risk areas for clinical trial success.
• Even approved drugs (like Relyvrio) can be withdrawn if follow-up trials disappoint.

The latest on in clinical trial failures in 2025:

Here’s a summary of the situation surrounding Sarepta’s Duchenne gene therapy, Elevidys:

Elevidys Safety Concerns & FDA Investigation

• In June 2025, the FDA launched an investigation after two non-ambulatory teenage patients died from acute liver failure within two months post-treatment with Elevidys (Fierce Pharma).
• A third death occurred in a 51-year-old enrolled in a separate limb-girdle muscular dystrophy gene therapy trial (SRP‑9004), which used the same viral platform (Fierce Biotech).

FDA Actions & Company Response

• The FDA requested that Sarepta voluntarily halt shipment of Elevidys; Sarepta initially refused, claiming no new safety risks for ambulatory patients, but later complied due to mounting pressure and to preserve regulatory relations (Reuters).
• Additionally, the FDA:
  • Placed all limb-girdle muscular dystrophy (LGMD) trials on clinical hold (Fierce Biotech).
  • Revoked Sarepta’s AAVrh74 platform technology designation due to safety concerns (U.S. Food and Drug Administration).
  • Is reviewing a possible full market withdrawal of Elevidys (Fierce Pharma).
• Sarepta plans to update Elevidys’ label with a liver toxicity warning, potentially a boxed warning, and explore using sirolimus to mitigate risks in future dosing (Fierce Pharma).

Impact on Sarepta

• Share price dropped 85–90%, declining from ~$15 billion to just over $1 billion in market capitalization (Barron’s, The Wall Street Journal, Reuters).
• Joe masses one strategic response:
  • Paused shipments effective July 22, after resisting initial FDA requests (Fierce Pharma).
  • Laid off ~500 employees (~36% of workforce) to save costs and support restructuring (Wikipedia).

Context & Implications

What’s Next

• Label update and risk mitigation: Sarepta is working with the FDA to add a black-box warning and adopt new liver safety protocols.
• Clinical and regulatory reviews: FDA continues reviewing Elevidys for possible withdrawal; global oversight includes Roche halting shipments in some regions (Reuters).
• Restructuring Sarepta: Ongoing pipeline review, cost cuts, and strategic shifts in response to FDA actions and safety concerns.

This development underscores growing regulatory scrutiny of gene therapies and emphasizes the crucial need for long-term safety data—even after approval. Sarepta is navigating a pivotal moment that may reshape treatment availability for Duchenne muscular dystrophy patients.

For More details: https://www.linkedin.com/pulse/safety-journey-tick-box-activity-rashmi-pant-0jqxf