FDA Official's LinkedIn Post Sparks Controversy Over Surrogate Endpoints and Lupus Drug
In a surprising turn of events, the pharmaceutical industry has been buzzing with controversy following a now-deleted LinkedIn post by Dr. George Tidmarsh, director of the FDA's Center for Drug Evaluation and Research (CDER). The post, which targeted the use of surrogate endpoints in drug approvals and specifically called out Aurinia Pharmaceuticals' lupus nephritis drug Lupkynis, has raised questions about the FDA's stance on approval processes and the future of certain approved medications.
Tidmarsh's Post Challenges Lupkynis Approval
Dr. Tidmarsh's LinkedIn post claimed that Lupkynis (voclosporin) carries "significant toxicity" and "has not been shown to provide a direct clinical benefit for patients." He further stated that for diseases such as lupus nephritis, companies have not conducted trials to demonstrate benefits on hard clinical endpoints like progression to end-stage renal disease.
The CDER director's comments have sent shockwaves through the industry, particularly because Lupkynis received full FDA approval in 2021 based on data showing higher renal response rates and quicker decline in urine protein creatinine ratio (UPCR) compared to standard-of-care treatment alone.
Aurinia Pharmaceuticals Responds
Aurinia Pharmaceuticals swiftly responded to Tidmarsh's claims, reaffirming their confidence in Lupkynis's risk-benefit profile. The company highlighted that the FDA approved an application for long-term use of Lupkynis in 2024, based on results from the Aurora 2 study, which demonstrated the drug's continued effectiveness over three years with a safety profile consistent with previous trials.
Following the controversial post, Aurinia's stock plummeted by 15.8% at market close on Monday, reflecting the immediate impact of Tidmarsh's comments on investor confidence.
Broader Implications for Drug Approvals
Dr. Tidmarsh's post has ignited a broader discussion about the use of surrogate endpoints in drug approvals. He announced that CDER would be evaluating surrogate endpoints used for FDA approval, citing "notable failures in confirmatory trials, such as those for exon skipping therapies in Duchenne muscular dystrophy (DMD)."
This statement has raised concerns for companies like Sarepta Therapeutics, whose exon-skipping DMD therapies could potentially face increased scrutiny. Analysts at Evercore ISI suggested that the odds of Sarepta's therapies being pulled from the market "just went up" in the wake of Tidmarsh's comments.
The pharmaceutical industry now awaits further clarification from the FDA regarding its stance on surrogate endpoints and the potential reevaluation of previously approved drugs. As the situation develops, it is clear that this controversy may have far-reaching implications for drug development, approval processes, and patient access to medications.
References
- FDA's Tidmarsh Singles Out Aurinia's Drug in LinkedIn Post About Surrogate Endpoints
Without naming Aurinia Pharmaceuticals, the CDER director in a now-deleted LinkedIn post claimed that for lupus nephritis, companies have not conducted post-approval studies “to demonstrate a benefit on hard clinical endpoints.”
- FDA official challenges safety, efficacy of Aurinia lupus med in now-deleted LinkedIn post targeting surrogate endpoints
A now-deleted LinkedIn post from a top FDA official has raised the hackles of Aurinia Pharmaceuticals as industry watchers puzzle over the official's decision to target the approvability of surrogate endpoints—and single out Aurinia’s lupus nephritis med Lupkynis—via social media.
Explore Further
What clinical data supports the long-term safety and efficacy of Lupkynis, and how does it compare to alternative treatments for lupus nephritis?
What are the key differences between surrogate endpoints and hard clinical endpoints in drug approval processes, and why are they controversial?
How might the FDA's potential reevaluation of surrogate endpoints impact currently approved drugs such as exon-skipping therapies for Duchenne muscular dystrophy?
What are the potential financial and market implications for Aurinia Pharmaceuticals if increased scrutiny on surrogate endpoints leads to further regulatory challenges for Lupkynis?
How does the use of surrogate endpoints in lupus nephritis drug trials compare to their use in other therapeutic areas, and what lessons can the industry learn from these findings?