Terms to Know About Obstructive HCM and CAMZYOS^®

Actin

A protein found in the heart muscle that works alongside myosin to help the heart contract and relax. Using a simple analogy: if myosin acts like people pulling on a rope, actin is the rope. Together, myosin and actin control how the heart muscle squeezes and releases with each heartbeat. CAMZYOS works by helping myosin let go of the actin, allowing the heart to relax more normally.

Alcohol Septal Ablation

A procedure that can help improve blood flow in the heart for some people with obstructive HCM. A small amount of alcohol is injected into a tiny artery that supplies the thickened heart muscle, causing that area of muscle to shrink over time. Along with septal myectomy, alcohol septal ablation is a type of surgery also known as septal reduction therapy. By removing the obstruction, surgery may also result in symptom relief.

Beta-Blocker

A type of prescription medicine often used to help manage symptoms of obstructive HCM, such as shortness of breath and fatigue. Beta-blockers are part of a group of traditional oHCM treatments that also includes calcium channel blockers and disopyramide. Most of these medications are used for symptom management of oHCM, but most are not approved by the FDA specifically for oHCM. Some data show that beta-blockers may also address the obstruction.

Calcium Channel Blocker

A type of prescription medicine that may be used to help manage symptoms of obstructive HCM. Calcium channel blockers are one of the traditional oral medication options used to manage symptoms of oHCM, along with beta-blockers and disopyramide. Most are not approved by the FDA specifically for oHCM.

CAMZYOS®

(mavacamten) is a prescription medicine used to treat adults with symptomatic obstructive HCM. CAMZYOS may improve your symptoms and your ability to be active.

Cardiac Myosin

A protein found in the muscle that makes up your heart. Myosin — along with another structure called actin — is responsible for making your heart muscle contract and relax, which helps your heart pump blood. In someone with obstructive HCM, too many myosin proteins are active at once, making it difficult for the heart muscle to relax. This causes the heart to work harder and use more energy.

Cardiac Myosin Inhibitor (CMI)

A class of medicine that works by reducing the overactivity of the cardiac myosin protein, helping the heart muscle relax and pump more normally. CAMZYOS is the first FDA-approved cardiac myosin inhibitor — a first-in-class treatment for symptomatic obstructive HCM. Unlike most medications commonly prescribed for oHCM, CAMZYOS was specifically designed to target myosin to treat the obstruction.

Cardiologist

A doctor who specializes in the diagnosis, prevention, and treatment of diseases of the heart. If you have been diagnosed with obstructive HCM, your care will likely be managed by a cardiologist.

Cardiomyopathy

A disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body. There are several types. Hypertrophic cardiomyopathy (HCM) is one of the most common inherited types and can affect the function of the heart.

Chest Pain

An uncomfortable feeling of pain, pressure, tightness, or discomfort in the chest that may occur with or without physical activity. Chest pain is one of the common symptoms of obstructive HCM. It often occurs during physical activity, when the heart has to work harder. If you are experiencing new or worsening chest pain, get medical help right away.

Disopyramide

A prescription medicine sometimes used alongside beta-blockers to help manage symptoms of obstructive HCM. Disopyramide is one of the traditional oral medications used for symptom management of oHCM, but is not approved by the FDA specifically for oHCM.

Dizziness

A feeling of unsteadiness, faintness, or being “off-balance.” Feeling light-headed is one of the common symptoms of obstructive HCM and may occur during physical activity when the heart is working harder. Dizziness is also the most common side effect of CAMZYOS treatment. Tell your healthcare provider if you experience dizziness or if it bothers you or does not go away.

Dyspnea

The medical term for shortness of breath — a feeling that it is hard to breathe or take a full breath. Dyspnea is one of the most common symptoms of obstructive HCM, especially during physical activity. If you’re still experiencing shortness of breath or fatigue on your current treatment, talk to your cardiologist. Symptom improvement may be possible.

Echocardiogram (Echo)

An echocardiogram (also known as an echo) is an imaging test, a type of ultrasound, that can be used to look at the heart and nearby blood vessels to see how well your heart is working. No radiation is involved in this procedure.

Echocardiographer

Also known as a cardiac sonographer, is a specialized medical professional who operates ultrasound technology to capture images of a patient’s heart and blood vessels. They play an important role in evaluating heart function and diagnosing obstructive HCM.

Electrocardiogram (ECG or EKG)

A quick test that records the electrical activity of the heart. Electrode patches are placed on the skin and measure how electrical signals move through the heart. An ECG can help detect irregular heartbeats and other changes sometimes seen in people with HCM.

Exercise Intolerance

Difficulty doing physical activity without experiencing symptoms such as shortness of breath, dizziness, or fatigue. Exercise intolerance is a common challenge for people with obstructive HCM. Improving the ability to be active is one important goal of treatment.

Exercise Stress Test

A test that shows how the heart responds during physical activity. You may walk on a treadmill or ride a stationary bike while your heart rate, blood pressure, and electrical activity are monitored. This test can help measure peak oxygen consumption (pVO₂) — the maximum amount of oxygen the body uses during exercise — which is one way doctors assess how obstructive HCM is affecting your ability to be active.

Fainting (Syncope)

A sudden, brief loss of consciousness. Fainting can be a symptom of obstructive HCM, especially during or after physical activity. It is also one of the most common side effects of CAMZYOS treatment. If you experience new or worsening symptoms, including fainting, get medical help right away.

Fatigue

A feeling of extreme tiredness or lack of energy that goes beyond normal tiredness. Fatigue is a common symptom of obstructive HCM and can make everyday activities feel more difficult. If you’re still experiencing fatigue on your current oHCM treatment, talk to your cardiologist — symptom improvement may be possible.

Functional Capacity

A term that describes a person’s ability to do physical activities in daily life. Improving functional capacity — meaning the ability to be more active without symptoms — is one of the goals of treating symptomatic obstructive HCM. CAMZYOS is indicated to improve functional capacity and symptoms in adults with symptomatic NYHA Class 2-3 obstructive HCM.

Genetic Testing

A blood or saliva test that looks for gene changes linked to hypertrophic cardiomyopathy. Because HCM is a heart disease that can be inherited — meaning it may run in your family due to genetics — genetic testing may be recommended for people with HCM and their close relatives. It is estimated that HCM impacts between 1 in 200 and 1 in 500 people.

HCM

Short for hypertrophic cardiomyopathy. See: Hypertrophic Cardiomyopathy.

Heart Failure

A serious condition where the heart cannot pump with enough force. The term “heart failure” does not mean that your heart has stopped — it means the heart is too weak to pump blood properly. Heart failure is not the same as a heart attack, cardiac arrest, or sudden death, but it is a serious condition that can lead to death if not addressed by a healthcare professional. When taking CAMZYOS, there is a risk of heart failure. The CAMZYOS REMS program is designed to help monitor your symptoms and side effects through required echocardiograms throughout treatment.

HOCM

Short for hypertrophic obstructive cardiomyopathy. Another common term for obstructive HCM that you may have heard in discussions with your doctor. See: Obstructive Hypertrophic Cardiomyopathy (oHCM).

Hypercontractility

When the heart muscle squeezes more forcefully than it should. In someone with obstructive HCM, too many myosin proteins are active at once, making the heart muscle hypercontractile — causing it to work harder and use more energy. This contributes to the thickening of the heart muscle and the blockage of blood flow.

Hypertrophic Cardiomyopathy (HCM)

A condition in which the heart muscle wall thickens and stiffens, making it harder for the heart to fill with blood. HCM is a heart disease that can be inherited, meaning it may run in your family due to genetics. It is estimated that HCM impacts between 1 in 200 and 1 in 500 people. When HCM causes the heart muscle to get so thick that it blocks or reduces blood flow from the heart to the rest of the body, it is called obstructive HCM. The majority of people with HCM have been diagnosed with obstructive HCM. If diagnosed with symptomatic NYHA Class 2-3 obstructive HCM, CAMZYOS may be prescribed.

KCCQ (Kansas City Cardiomyopathy Questionnaire)

A questionnaire used in clinical studies to measure how a heart condition affects a person’s daily life. It asks about symptoms, physical limitations, and overall quality of life. The KCCQ was used in CAMZYOS clinical studies to help measure how patients felt and functioned during treatment. Your doctor may use similar questionnaires to track how your symptoms are changing over time.

Left Ventricle

The main pumping chamber of the heart. The left ventricle pumps oxygen-rich blood out to the rest of the body. In obstructive HCM, the walls of the left ventricle become thickened, making it harder for the heart to fill with and pump blood normally.

Left Ventricular Outflow Tract (LVOT)

The pathway blood takes as it leaves the left ventricle — the heart’s main pumping chamber — and travels to the rest of the body. In obstructive HCM, the thickened heart muscle can narrow or block this pathway, making it harder for blood to flow freely. The degree of this blockage is measured by the LVOT gradient.

LVOT Gradient

A measurement of the pressure difference across the left ventricular outflow tract (LVOT) — the pathway blood uses to leave the heart. Obstruction is measured by post-exercise LVOT gradient. A higher LVOT gradient means there is more blockage to blood flow. A lower LVOT gradient means more blood is able to flow freely from the heart. Doctors use this measurement to understand how serious the obstruction is and to monitor how well treatment is working.

Mavacamten

The generic (non-brand) name for CAMZYOS. Mavacamten is a prescription cardiac myosin inhibitor — the first FDA-approved medicine of its kind — used to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). See also: Cardiac Myosin Inhibitor.

Mitral Valve

One of four valves in the heart, located between the left atrium and the left ventricle. In some people with obstructive HCM, a part of the mitral valve can be pulled toward the outflow tract during a heartbeat — a movement called systolic anterior motion (SAM) — which can worsen the obstruction of blood flow.

Myosin

A protein found in the muscle that makes up your heart. Myosin — along with another structure called actin — helps your heart muscle contract and relax, which is how your heart pumps blood. Think of it this way: myosin acts like people pulling on a rope. When enough people pull, your heart muscle contracts. When they take a break, your heart muscle can relax. In someone with obstructive HCM, too many myosin proteins are active at once — too many people pulling on the rope — making it difficult for the heart muscle to relax. CAMZYOS works directly with the myosin, helping more of them take a break from pulling. This allows your heart to relax.

NYHA Classification (New York Heart Association Functional Classification)

A system doctors use to describe how much a heart condition is affecting a person’s daily activities and quality of life. There are four NYHA classes:

Class 1: No symptoms during ordinary physical activity
Class 2: Mild symptoms during moderate physical activity
Class 3: Noticeable symptoms during minimal physical activity; comfortable at rest
Class 4: Symptoms even at rest

CAMZYOS is approved to treat adults with symptomatic NYHA Class 2-3 obstructive HCM.

Obstruction

A blockage or narrowing that limits blood flow. In obstructive HCM, the thickened heart muscle blocks or reduces blood flow from the heart to the rest of the body. The degree of obstruction is measured by the LVOT gradient. CAMZYOS was specifically designed to target myosin to reduce the obstruction.

Obstructive Hypertrophic Cardiomyopathy (oHCM)

A type of hypertrophic cardiomyopathy (HCM) in which the thickened heart muscle blocks or reduces blood flow from the heart to the rest of the body. When this causes symptoms — such as shortness of breath, fatigue, chest pain, or feeling light-headed — it is called symptomatic obstructive HCM. Also referred to as HOCM. CAMZYOS is a prescription medicine used to treat adults with symptomatic obstructive HCM that may improve your symptoms and your ability to be active.

OHCM

Short for obstructive hypertrophic cardiomyopathy. See: Obstructive Hypertrophic Cardiomyopathy (oHCM).

Palpitations

An unusual awareness of your own heartbeat — often felt as a racing, fluttering, or pounding sensation in your chest. Palpitations are a common symptom of obstructive HCM. A racing heart is also one of the symptoms to watch for that may signal heart failure. Get medical help right away if you experience new or worsening palpitations.

Placebo

An inactive treatment used in clinical studies that contains no medicine. In a clinical study, some participants receive the medicine being tested while others receive a placebo. This allows researchers to accurately measure the true effect of the medicine. In the CAMZYOS studies, CAMZYOS was compared against a placebo to measure its effectiveness in reducing obstruction and improving symptoms in adults with symptomatic obstructive HCM.

Prior Authorization

A Prior Authorization (PA) is a verification from your health care provider (HCP) that states that your prescription is medically necessary. Your health insurance provider may require a PA before they will cover your prescription. Your doctor submits information to your insurance company explaining why the medicine is medically necessary for you. If your prior authorization is denied, there are resources available to help. There are resources available to help you understand your rights after a PA denial, how to navigate next steps and learn more about how your health care provider may be able to support you at the camzyos.com website.

Provocation (Provocative Testing)

A method used during echocardiography to reveal the full degree of obstruction in the heart. In some people with obstructive HCM, the blockage of blood flow may not be fully visible when the heart is at rest — but can be “provoked” or revealed during physical activity or certain maneuvers. Common provocative methods include exercise stress testing and the Valsalva maneuver. Provocative testing helps your cardiologist get a more accurate measurement of your LVOT gradient and better understand how obstructive HCM is affecting your heart.

Pumping Blood (How the Heart Pumps Blood)

The heart is a muscle that works like a pump, moving blood throughout your body with every beat. With each heartbeat, the heart contracts (squeezes) to push blood out, then relaxes to fill back up. The left ventricle — the heart’s main pumping chamber — sends oxygen-rich blood to the rest of the body. In obstructive HCM, the heart muscle becomes thickened and stiff, making it harder for blood to flow freely.

PV02 (Peak oxygen uptake)

Measures the maximum amount of oxygen the body uses during strenuous exercise and is considered a key indicator of heart health.

REMS (Risk Evaluation and Mitigation Strategy)

A safety program required by the FDA for certain medicines. CAMZYOS is only available through the CAMZYOS REMS program because in some patients, CAMZYOS can cause the heart to become too relaxed, increasing the risk of heart failure. The program requires regular echocardiograms to monitor heart health during treatment and screens for drug interactions. Both patients and healthcare providers must be enrolled, and CAMZYOS is only dispensed through REMS-certified specialty pharmacies.

SAM (Systolic Anterior Motion)

A movement of the mitral valve that can occur during a heartbeat in some people with obstructive HCM. When part of the mitral valve moves toward the outflow tract, it can worsen the blockage of blood flow. SAM can be detected on an echocardiogram.

Septal Hypertrophy

Abnormal thickening of the septum — the wall between the two main pumping chambers of the heart. This thickening is a hallmark feature of hypertrophic cardiomyopathy and can cause or worsen the obstruction of blood flow.

Septal Reduction Therapy

A category of surgical procedures used to reduce the thickness of the heart muscle and improve blood flow in people with obstructive HCM. There are two main types: septal myectomy (surgical removal of part of the thickened muscle) and alcohol septal ablation (a less invasive procedure using alcohol to shrink the thickened area). When the obstruction is severe, or medication fails to keep oHCM from worsening, surgery may be an option. By removing the obstruction, surgery may also result in symptom relief.

Septum

The wall of muscle that divides the left and right sides of the heart. In hypertrophic cardiomyopathy, this wall often becomes abnormally thick — a condition called septal hypertrophy — which can block blood flow out of the heart.

Shortness of Breath

A feeling that it is hard to take a full breath or that breathing takes more effort than usual. Shortness of breath is one of the most common symptoms of obstructive HCM, especially during physical activity. If you’re still experiencing shortness of breath on your current oHCM treatment, you don’t have to wait for your symptoms to get worse before talking to your cardiologist. With CAMZYOS, symptom improvement and the ability to be active may be possible.

Specialty Pharmacy

A pharmacy that dispenses specialty medications — including medicines that require special handling, monitoring, or safety programs. Because of the requirements of the CAMZYOS REMS program, CAMZYOS is only available through REMS-certified specialty pharmacies. CAMZYOS is not available at local retail pharmacies. Your doctor will give you information on how to receive your CAMZYOS medication from a specialty pharmacy, which will send your medication directly to your home.

Symptomatic Obstructive HCM

Obstructive HCM that causes noticeable symptoms — such as shortness of breath, rapid heartbeat, chest pain, feeling light-headed, fainting, or fatigue — that affect everyday life. Obstructive HCM can progress over time and lead to other serious conditions, so it’s important to keep track of your symptoms and keep your doctor informed. CAMZYOS is indicated for the treatment of adults with symptomatic NYHA Class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.

Syncope

The medical term for fainting, it is one of the most common side effects of CAMZYOS.

Systolic Dysfunction

A condition in which the heart muscle becomes too weak or too relaxed to pump blood with enough force. In some patients, CAMZYOS can cause the heart to become too relaxed, which increases the risk of systolic dysfunction and heart failure. This is why the CAMZYOS REMS program requires regular echocardiograms — to monitor for any signs of systolic dysfunction during treatment.

Thickened Heart Walls

A hallmark feature of hypertrophic cardiomyopathy (HCM). In HCM, the heart muscle wall thickens and stiffens, making it harder for the heart to fill with blood. When this thickening becomes so significant that it blocks or reduces blood flow from the heart to the rest of the body, it is called obstructive HCM. Thickened heart walls can be seen and measured on an echocardiogram.

Valsalva Maneuver

A breathing technique where a person bears down or strains, sometimes used during echocardiography. Your doctor may ask you to perform the Valsalva maneuver to help reveal the full degree of obstruction in the heart. The LVOT gradient may increase during this maneuver, helping your cardiologist get a more accurate measurement.