A History of Pazopanib
There are no FDA-approved therapeutics for the treatment of HHT. Cure HHT sought to change that.
In many ways, the history of pazopanib is not unlike the story of the HHT community. Both are defined by persistence: a long battle for awareness, funding, and ultimately, solutions. Now, after years of dedicated effort, the paths of pazopanib and HHT have converged, moving forward in unison and resulting in the accomplishment of a pazopanib clinical trial filling and closing to enrollment. This is a major step forward for the future of HHT treatment, as we push to bring our community FDA approved therapies for the first time.
Here’s a look back at how we got here.
WHAT IS PAZOPANIB?
The story of pazopanib starts more than 15 years ago. In 2009, the pharmaceutical giant GlaxoSmithKline secured FDA approval to use the drug, marketed under the name Votrient™, for treatment of patients with renal cell carcinoma — a rare kidney cancer. Votrient functions as an angiogenesis inhibitor, meaning it helps to prevent the formation and growth of new blood vessels.
When pazopanib was approved to treat kidney cancer, it arrived at a turning point in care—when doctors were shifting from older immune-based treatments to newer drugs that stop blood vessels from feeding tumors. But this wasn’t the only way pazopanib would change how diseases
are treated.
A NEW APPROACH
By 2015, the success of pazopanib in cancer treatment had sparked new interest in what else the drug might help. Cure HHT assembled a team led by Dr. Marie Faughnan to research whether it could reduce bleeding in people with HHT. Their theory was that since pazopanib prevented the growth of new blood vessels, it might also prevent the formation of blood vessel tangles known as arteriovenous malformations (AVMs) or telangiectasias –hallmarks of HHT and the cause of HHT-related bleeding. Several HHT Centers were already using the drug off label with low dosages and seeing positive results with limited side effects.
Dr. Faughnan and several HHT Center physicians started small, enrolling seven HHT patients in a multi-center project to observe how pazopanib impacted symptoms and quality of life. The results were promising. Over the course of the trial, most patients showed meaningful improvements in both nosebleed duration and severity.
Encouraged by these early efforts, Dr. Faughnan and her study colleagues recommended further and larger-scale research on the potential use of pazopanib as a treatment for HHT bleeding. For a moment, the future looked bright.
But there was a problem.
In the US, drugs must be approved by the Food & Drug Administration (FDA) before they can be marketed and sold for the treatment of a specific condition. That approval hinges on the results of clinical trials, most of which are funded by large drug manufacturers.
When the multi-site trial began, GlaxoSmithKline still owned pazopanib under the name Votrient™. But by March of 2015, GSK had sold much of its oncology portfolio — pazopanib included — to the manufacturer Novartis. And despite the promising results from the initial seven-person study, Novartis declined to sponsor the further trials needed to secure FDA approval for the drug as an HHT treatment.
A BOLD IDEA
HHT, unlike cancer, receives significantly less research funding. While cancer research garners billions annually—one estimate suggesting over $24 billion between 2016 and 2020—HHT remains underfunded. This disparity highlights the critical need to advocate for rare diseases.
Although HHT may not be as prevalent as cancer, it profoundly impacts patients’ and families’ quality of life. These individuals deserve access to optimal care.
When Novartis declined to pursue promising research that could transform HHT treatment, it put the HHT community at a crossroads. With opportunities like this few and far between, Cure HHT needed to act swiftly and boldly.
A COMMUNITY EFFORT
Since drug owners and manufacturers are responsible for trials, we decided we couldn’t just sit on the sidelines. We had to own and manufacture pazopanib — the potential benefits this drug could have on our community was too great. With the support of our remarkable community, we raised nearly $1 million toward our goal and began advocating for additional funding through the Department of Defense and the FDA.
And our efforts paid off. In 2022, we received a $5.2 million grant from the DoD and an additional $800,000 from the FDA to support a trial studying the use of pazopanib as a treatment for HHT. The FDA also awarded us with a Breakthrough Therapy designation, a process that accelerates the review of drugs that have the potential to treat serious conditions more effectively than available therapies.
Since then, Cure HHT has been working tirelessly to build the structure for our clinical research efforts: funding studies, collecting and analyzing data, building protocols and collaborating with partner organizations across statistics, device manufacturing, laboratories, auditing, regulation, and more. We are one of the few nonprofit organizations in the world to be directly running a trial like this. We are relentless in our fight to bring this community better treatment and care.
Now we are one step closer to doing just that, as we announce that our clinical trial has completed enrollment in early 2025! If the results of our trial are successful, we hope to secure FDA approval of pazopanib as a dedicated therapeutic for the treatment of HHT.
This would make pazopanib one of the first FDA-approved HHT therapeutics, providing new care options for those impacted by this disease. FDA-approved treatments are not only much easier to cover through insurance, they’re also much more
broadly accessible.
Today, the power to deliver affordable, life-changing care is in our hands — and we won’t stop until it’s in yours.
This story is featured within the 2025 Spring / Summer Newsletter, now available digitally. Find important resources, announcements, programs, stories from staff, community updates, and more. Download your copy today.