Medicus Pharma (NASDAQ:MDCX) CEO Raza Bokhari talked with Proactive about the company’s recent steps to expand compassionate access to Skinject, its lead non-invasive treatment for non-melanoma skin diseases.
The initiative involves a partnership with the Gorlin Syndrome Alliance, aiming to address unmet needs for patients living with this rare genetic condition.
Bokhari explained that Gorlin syndrome affects around 1 in 31,000 individuals worldwide and often results in a lifelong burden of basal cell carcinoma.
The company is working toward FDA compassionate use approval for Skinject and sees a strategic alignment between its innovation, regulatory progress, and patient advocacy.
Bokhari highlighted the FDA’s current openness to advancing treatments for rare diseases and noted that the company is engaging key supporters in Washington, including board member Cathy McMorris Rodgers, to help progress the application.
If successful, this move would enable select patients to access Skinject outside of formal clinical trials while still being monitored for safety.
Proactive: Welcome back inside our Proactive newsroom. And joining me now is Dr. Raza Bokhari. He is the CEO of Medicus Pharma. Dr. Bokhari, good to see you again. How are you?
Raza Bokhari: I'm doing really well. Thank you so much for having me back on your program.
The company is out with a news release talking about partnering with the Gorlin Syndrome Alliance to expand compassionate access to Skinject. Let's take a step back and talk about Gorlin syndrome and how that connects to your work.
I really appreciate this opportunity to speak about this and create awareness around this rare disease called Gorlin syndrome. It's a genetic, autosomal dominant illness, affecting 1 in 31,000 people globally. Around 11,000 individuals live with Gorlin syndrome and a normal lifespan, but it’s a very difficult life — many would say torturous — due to lifelong cancer, particularly basal cell carcinoma. They may end up having hundreds of these cancers during their life.
It intersects with our work at Medicus Pharma, where our lead asset, Skinject, is a noninvasive treatment for non-melanoma skin diseases, particularly basal cell carcinoma. The traction we're building is generating attention, and the Gorlin Syndrome Alliance reached out to us to explore whether we could work together for expanded or compassionate use approval by the FDA. We’re very excited that this could potentially provide a treatment to those with no current alternatives.
And as you push this toward the FDA, having that group on your side—representing patients and telling their stories—must be helpful?
Absolutely. This is a perfect alliance of innovative research, regulatory leadership, and patient advocacy, serving an unmet need for a rare disease that currently has no available treatment due to genetic mutation. The FDA is increasingly sensitive to unmet needs and is interested in accelerating therapies, so the timing is promising.
Just last week, the FDA commissioner released a list of ten rare drugs that could be fast-tracked. Our hope is that, through our board member Cathy McMorris Rodgers—former congresswoman and chair of Energy and Commerce—we might gain support in Washington to help advance and possibly get on that list. But time will tell. What's important is that we offer hope to Gorlin syndrome patients, not just in the U.S. but globally.
If granted, this would allow patients to access treatment outside clinical trials, right?
That’s our understanding — that it would be approved for a select group of patients under compassionate use. Some conditions must be met, like collecting all safety data from our patch. That’s where the Gorlin Syndrome Alliance is a great partner. It’s run by patients and families, and they maintain a registry and coordinate with over 11,000 patients in the U.S. They also have global connections.
They are not only aligned with us in wanting this treatment available but can also help us reach those who may need it. We are thrilled our work may make a difference at the most compassionate level.
Quotes have been lightly edited for clarity and style