"Confirmed long-term Tx effects of Camzyos…new oHCM Tx"

Dr. Neal K. Lakdawala, Professor at Brigham and Women's Hospital in the United States

Interim analysis results at week 18 show maintained efficacy·safety

"Introduction of targeted treatment for oHCM…oral drug that reduces the number of required surgical treatment"

Dr.

 

Neal K.

 

Lakdawala, Professor at Brigham and Women

"In a clinical study spanning three years, Camzyos has demonstrated consistent patient symptom management and safety in many patients with obstructive hypertrophic cardiomyopathy (oHCM).

 

Given its proven efficacy in long-term treatment, it is expected that many patients with oHCM can significantly improve their quality of life through this treatment without the need for surgical treatment." During a recent meeting with Daily Pharm, Dr.

 

Neal K.

 

Lakdawala, Professor at Brigham and Women's Hospital in the United States, remarked on 'Camzyos,' a first-in-class targeted treatment option for obstructive hypertrophic cardiomyopathy (oHCM), as above oHCM is a type of hypertrophic cardiomyopathy (HCM) characterized by abnormal heart muscle thickening, primarily in the left ventricle.

 

This condition obstructs blood flow and impairs heart function.

 

Common symptoms include shortness of breath, chest pain, and fainting, and it can lead to complications such as heart failure, atrial fibrillation, and even sudden cardiac death.

 

Until now, treatment methods besides surgical treatment because no treatments could directly target pathophysiological causes of oHCM.

 

While unmet needs for oHCM have been high, the introduction of Camzyos as the first treatment targeting the pathophysiology of the disease has opened the gate for new treatment options.

 

Camzyos is a treatment for oHCM that reduces excessive cross-bridge formation between actin and myosin in the heart muscle, which is the underlying cause of the condition.

 

This mechanism helps relax the overly contracted heart muscle.

 

Additionally, the therapy has demonstrated myocardial remodeling effects, improving not only the function of the heart but also its structural integrity.

 

Camzyos can be reimbursed with the national health insurance starting this month in South Korea.

 

Now, Korean patients with oHCM can use targeted treatment options besides the option of surgical treatment.

 

Dr.

 

Lakdawala remarked that Camzyos could change the treatment paradigm for oHCM since the drug's efficacy and safety were demonstrated not only in clinical trials for approval but also in long-term administration studies.

 

Camzyos demonstrates consistent effectiveness in long-term administration studies In the EXPLORER-HCM trial, which was the basis for approval, Camzyos demonstrated significant improvement in the primary endpoint, which assessed both symptom severity (NYHA class) and exercise capacity (pVO2), compared to the placebo group.

 

Camzyos continues to establish additional evidence for its long-term safety and efficacy.

 

At the European Society of Cardiology annual congress (ESC 2024) in September, results from the MAVA-LTE clinical study evaluating Camzyos' long-term effects and safety were unveiled.

 

Interim results showed that at week 180 of treatment, 66.3% of patients achieved NYHA Class I, with 46.8% demonstrating a complete response (NYHA Class I+Valsalva LVOT pressure gradient of ≤30 mmHg).

 

The NYHA classification system, established by the New York Heart Association, evaluates the extent of symptoms and activity limitations in patients.

 

Additionally, N-terminal pro-brain natriuretic peptide (NT-proBNP) levels decreased by 81% from baseline at week 180, with 53.8% of patients reaching normal NT-proBNP levels.

 

NT-proBNP serves as a biomarker for assessing heart failure severity and prognosis, often elevated under conditions of excessive myocardial stress or stretch in heart failure patients.

 

The EQ-5D-5L score, which assesses health-related quality of life, showed improvement, indicating better subjective health perceptions among patients.

 

"Camzyos' long-term data, confirmed through the MAVA-LTE study, shows the positive potential of the drug.

 

Patients' symptoms have been consistently maintained well during 3 years of long-term study, and the safety profile has also been consistent," Dr.

 

Lakdawala emphasized.

 

"In fact, the number of patients choosing to undergo surgical treatments is declining after the introduction of Camzyos.

 

The Real-World Data of Camzyos confirmed in clinical practices show an equivalent level of effectiveness and safety profile to results demonstrated in previous clinical studies," Dr.

 

Lakdawala added.

 

"Although the outcomes may vary depending on individual's pathophysiology, most patients undergone Camzyos treatments also have significantly improved symptoms or show much improved responses." "Camzyos' myocardial remodeling effect, which improves cardiac function and structure, has gained most attention since the early EXPLORER-HCM study," Dr.

 

Lakdawala stated.

 

"The study results showed significant reductions in key indicators of cardiac hypertrophy in HCM, such as left ventricular wall thickness and left ventricular mass, following Camzyos treatment.

 

These changes were observed as early as 24 to 30 weeks into the treatment and were sustained over the long-term treatment period," he added.

 

Despite the introduction of targeted treatment option..undiagnozed patients↑ Despite the recent introduction of targeted treatment option, diagnosing oHCM is still challenging.

 

According to Dr.

 

Lakdawala, many cardiomyopathy patients often adapt to their long-standing symptoms, and they fail to recognize their condition.

 

Typically, as symptoms gradually restrict physical activity, patients adjust to a limited lifestyle and forget that their health status is not normal.

 

Dr.

 

Lakdawala explained that many patients only realize that HCM caused their past symptoms after receiving a diagnosis and experiencing improvement following treatment.

 

"The issue of undiagnosed HCM is one of the major global challenges today.

 

To address this issue, it is crucial to educate cardiology healthcare providers through multiple channels to help them understand that patients’ symptoms may be associated with this condition," Dr.

 

Lakdawala emphasized.

 

"When there is a thickening of the left ventricular wall, it is important not to simply attribute it to secondary effects of hypertension or overlook the severity of the condition.

 

Additionally, the lack of appropriate imaging diagnostics, which often leads to missed diagnoses of HCM, is another critical issue that must be resolved," he stressed.

 

With the introduction of Camzyos, patients' left ventricular outflow obstruction and related symptoms can be improved by taking an oral drug once a day.

 

While surgical treatment is still necessary for some patients, most are expressing satisfaction that their symptoms can be effectively managed through oral medication alone, according to Dr.

 

Lakdawala.

 

"Recent guidelines for HCM in the United States and Europe prioritize a patient-centered approach above all," he stated.

 

"These guidelines focus on symptom management as the primary goal and emphasize expanding treatment options to improve symptoms.

 

Previously, surgical treatments like septal reduction therapy (SRT) had been the primary option.

 

Introducing Camzyos in the guidelines provided a new option, expanding patients' treatment choices." Dr.

 

Lakdawala added, "In the past, HCM patients were often prohibited or restricted from engaging in physical activity.

 

However, recent studies have confirmed that appropriate levels of exercise are safe for these patients.

 

Based on this evidence, we are gradually relaxing guidelines to permit regular exercise and moderate physical activity, while continually seeking ways to enhance the quality of life for patients with HCM."