Cardiol Therapeutics (TSX:CRDL, NASDAQ:CRDL) made headlines earlier in the week when it revealed that the US Food and Drug Administration had granted its lead drug candidate CardiolRx orphan drug designation, indicating potential efficacy in treating recurrent pericarditis.
The designation provides benefits such as marketing exclusivity and fee reduction, and the company's share prices have surged as a result.
Cardiol's focus on addressing the symptoms of recurrent pericarditis, including chest pain and reduced quality of life, underscores its commitment to improving patient outcomes in this debilitating condition.
In this exclusive interview with CEO David Elsley, he sheds light on the company's extensive global program addressing acute myocarditis, a severe form of heart inflammation, and the MAvERIC-Pilot study, targeting recurrent pericarditis. Elsley discusses the implications of this designation, expressing confidence in Cardiol's development efforts and its commitment to addressing unmet medical needs in rare diseases.
Proactive: Let’s start with getting some background on Cardiol’s lead drug candidate, CardiolRx.
DE: We are developing CardiolRx for two very important heart diseases that affect a younger patient population. Acute myocarditis is our largest program. This is a devastating form of heart muscle inflammation, and a leading cause of sudden cardiac death in people under the age of 35. We're running one of the largest programs ever conducted in the past 30 years. It's a global program that’s tracking well ahead of schedule.
More important, though, is the MAvERIC-Pilot study, which is in recurrent pericarditis, which is another devastating form of inflammation affecting a younger age population. Some of the patients suffer chest pain and shortness of breath that require very powerful interventions, which can have toxicology and immunosuppressive concerns. They’re also very costly.
What does it mean to have CardiolRx granted orphan drug designation?
This is a very significant development for the company. Pericarditis is a condition that affects about 160,000 Americans and similar populations in other regions of the world, and about 40,000 of those patients don't have good treatment options. If they don't respond to mild non-steroidal therapies, they go on a path where they either have to endure the toxicology of steroids or go on powerful biologics that can be immunosuppressive, increasing infection risk and are costly and not easily accessible.
What the FDA embarked upon many years ago is the development of this orphan drug designation program to incentivize sponsors to develop drugs for important rare diseases that aren't well served and are well treated. They award sponsors and drug developers periods of exclusivity that can range from seven to nine years in the United States and upwards of 10 years in certain regions of Europe. This also speeds the path to regulatory designation, providing a path to fast-track designation, and significantly reduces drug filing fees and registration fees. So, it's a real wind in the sails for drug development to put a focus and put a spotlight on rare conditions that destroy people's lives.
In the case of recurrent pericarditis, this affects people in their prime of life, typically in their 30s and 40s. They're generally athletic and healthy. This condition, affecting the lining around the heart, creates friction, chest pain, and shortness of breath that is truly debilitating. With this orphan drug designation expediting our development path, we have great hopes that our therapy could potentially become a new first-line treatment option for thousands of patients who currently lack adequate options.
Does this designation from the FDA serve as a vote of confidence that to say, essentially, you're on the right track?
Absolutely. As part of this designation, we were requested to submit initial clinical data from the ongoing MAvERIC-Pilot study, led by esteemed institutions such as the Cleveland Clinic, Mayo Clinic, and Mass General Harvard, with several other prominent US centers recently joining. This package of information provided to the Food and Drug Administration, in the context of this crucial decision in favor of Cardiol, included the initial clinical data filed confidentially. This signifies significant support for our development efforts and marks a momentous day for pericarditis patients as we accelerate the introduction of a crucial new drug treatment for this underserved population.