A Heartfelt Solution: Camzyos

Pharmaceutical ExecutivePharmaceutical Executive: September 2023
Volume 44
Issue 9

First-of-its-kind treatment offers hope in the fight against rare heart disease.


In April of 2022, FDA approved Bristol Myers Squibb’s (BMS) Camzyos (mavacamten), the first treatment to target and inhibit cardiac myosin, the underlying protein involved in hypertrophic obstructive cardiomyopathy (HOCM), a rare disease that thickens and hardens the heart muscle wall—ultimately resulting in an obstruction of blood flow from the heart to the rest of the body.

For patients, suffering with HOCM means battling shortness of breath, palpitations, and a drastically diminished quality of life, progressing toward life-threatening complications over time or via physical exertion. According to BMS’ website, it’s estimated that hypertrophic cardiomyopathy (HCM) impacts between one in 200 and one in 500 people, with HOCM being the most common type.

High unmet need

Pharmaceutical Executive® spoke with Tom Garner, senior vice president, US cardiovascular and established brands, BMS. Prior to his promotion to his current role, Garner oversaw the US approval and launch of Camzyos. The orphan drug, originally developed by MyoKardia Inc., was a key asset in BMS’ 2020 acquisition of the California-based biotech for $13.1 billion.

Tom Garner

Tom Garner

“Historically, there was a reliance on beta blockers and calcium channel blockers dictated solely by empirical evidence…‘This is what we have, so we’ll use it.’ This meant constantly increasing dosages as symptoms worsened—shortness of breath, fatigue, palpitations—as patients’ lives diminished, becoming much less active,” says Garner.

As symptoms worsened with HOCM, the only other options that were available were a catheter-based procedure or open-heart surgery, both of which can come with significant complications for an HCM patient.

“Camzyos represents a groundbreaking shift, being the first targeted therapy addressing the root cause of HOCM, as opposed to just managing symptoms,” adds Garner.

For BMS, offering a simple oral capsule that targets the root cause of this serious cardiovascular condition underscores its commitment to enhancing patient outcomes in areas with high unmet needs and further expands on the company’s history and involvement in addressing cardiovascular disease. BMS has been a pacesetter in the cardiovascular field for the last 60 years, with drugs such as Eliquis and Plavix under its belt. Camzyos is the latest addition to this lineup.

The launch

In approaching an area without any treatments beyond reducing symptom burden, there really isn’t a launch playbook; as such, Garner and his team intensively studied the patient journey as well as treatment avenues to truly understand the cardiologist’s perspective. Once they determined the patient flow, they began to craft and define their market strategy.

Given the complexity of the disease and recognizing that patients tend to gravitate toward major HCM centers, they strategically mapped out the centers and strengthened alliances with thought leaders and key players, ensuring that those who were caring for the patients were well-versed, informed, and understood every nuance about the diagnosis and treatment. They also formed and grew relationships with patient societies like the Hypertrophic Cardiomyopathy Association as well as professional cardiovascular giants like the American Heart Association and the American College of Cardiology.

Risk Evaluation and Mitigation Strategy

In conjunction with FDA and as required, Garner and his launch team integrated a Risk Evaluation and Mitigation Strategy (REMS) into their strategy for Camzyos. Recognizing the safety concerns associated with potential heart failure risks, especially if dosed incorrectly, REMS was instituted to strike a balance between providing patients with access while ensuring judicious use and monitoring. Prescribers must be well-informed about the drug’s intended use and its risks, strictly adhering to its label and recommended dosing.

Notably, Camzyos was the first cardiovascular drug in nearly a decade to be introduced with a REMS drug safety program, highlighting its distinctive position in the market. It’s a nice balance of offering a transformative medication while diligently addressing its safety profile.

Patient support

After being prescribed Camzyos and enrolling in the “MyCam-zyos” patient support program, each patient is paired with a dedicated “nurse navigator” to assist throughout their treatment journey. In addition, they’ll collaborate with a patient access specialist to help with insurance details, understand out-of-pocket expenses, and explore available resources.

Moving forward

BMS’ introduction of Camzyos showcases not just a novel solution for HOCM patients but an example of a strategic product launch. BMS navigated the intricacies of launching in an undefined disease space. The company hopes its multi-faceted strategy, balancing innovative treatment with rigorous safety protocols, will position the treatment as a key game changer for years to come. Commercially, early signs point to promise for the projected future blockbuster. Camzyos posted $75 million in sales in the first half of 2023.

Up next for the treatment is a hopeful indication for non-obstructive HCM, which affects about 30% of those with HCM, according to Garner. The condition results in heart muscle thickening but without the physical blockage/obstruction of blood flow from the left ventricle. But symptoms like shortness of breath, chest pain, and fatigue still occur.

“In all my years and the numerous product launches I’ve overseen, the immediate and profound impact on patients with this particular product stands out,” says Garner. “The many personal stories we hear from patients are both humbling and deeply gratifying. To know that we’re making a difference in their lives—enabling them to enjoy simple joys like walking their dogs or playing with their grandchildren—is the very essence of why we do what we do.”

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