RegulationOrphan Drug (United States), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (South Korea), Breakthrough Therapy (United States)

Structure/Sequence

Molecular FormulaC15H19N3O2

InChIKeyRLCLASQCAPXVLM-NSHDSACASA-N

CAS Registry1642288-47-8

Clinical Trials associated with Mavacamten

NCT06856265

/ Not yet recruitingNot ApplicableIIT

Efficacy of Mavacamten Combined With Radiofrequency Ablation in Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This study aims to evaluate the efficacy and safety of Mavacamten combined with radiofrequency ablation compared to Mavacamten alone in patients with symptomatic obstructive hypertrophic cardiomyopathy (HOCM).
Participants were randomized into two groups:

Start Date26 Feb 2025

Sponsor / Collaborator

Shanghai Chest Hospital

NCT06146660

/ RecruitingNot Applicable

CAMZYOS® (Mavacamten) Post-Marketing Surveillance in Korean Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM)

The purpose of this observational post-marketing surveillance study is to assess the real-world safety of mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adult participants in Korea. Participants who will receive at least 1 dose of mavacamten will be enrolled.

Start Date24 Jul 2024

Sponsor / Collaborator

Bristol Myers Squibb Co.

NCT06338202

/ RecruitingNot Applicable

Real-World Effectiveness of MAVacamtEN in Canada (MAVEN): A Retrospective Physician Survey of Patient Charts

The purpose of this observational, retrospective, physician survey study is to describe the real-world effectiveness and use of mavacamten in participants who initiated treatment for obstructive hypertrophic cardiomyopathy (oHCM) in Canada.

Start Date26 Jun 2024

Sponsor / Collaborator

Bristol Myers Squibb Co.

100 Clinical Results associated with Mavacamten

100 Translational Medicine associated with Mavacamten

100 Patents (Medical) associated with Mavacamten

354

Literatures (Medical) associated with Mavacamten

01 Jun 2025·CURRENT PROBLEMS IN CARDIOLOGY

A Multidimensional Approach to Understanding Genetic Diversity, Risk Stratification, and Personalized Interventions in Pediatric Hypertrophic Cardiomyopathy

Review

Author: Alshabrmi, Fahad M ; Al-Doaiss, Amin A ; Alwanian, Wanian M ; Alromaihi, Mona ; Thornbury, William ; Alfifi, Somayah Mohammad ; AlDuayji, Nouf Nasser ; Alrumaihi, Faris ; Khan, Shahid Ullah ; Alharbi, Hajed Obaid

Hypertrophic cardiomyopathy (HCM) in children presents unique challenges distinct from adult manifestations, with potentially devastating consequences, including sudden cardiac death. This comprehensive review synthesizes current evidence on the pathophysiology, clinical presentation, and management of pediatric HCM, highlighting critical differences from adult populations. While affecting approximately 1 in 500 individuals, pediatric HCM demonstrates more significant etiological heterogeneity, with up to 35% of cases stemming from non-sarcomeric causes, including RASopathies, metabolic disorders, and syndromic conditions. This etiological diversity contributes to variable disease trajectories and treatment responses, creating a significant research gap in pediatric-specific management protocols. Current pharmacological approaches primarily employ beta-blockers as first-line therapy, with calcium channel blockers serving as alternatives for intolerant patients. However, these conventional medications manage symptoms without addressing underlying pathophysiology or preventing disease progression. Emerging investigational therapies, including angiotensin receptor blockers and myosin inhibitors like mavacamten, show promise in preliminary studies but lack robust pediatric-specific evidence. Surgical interventions, including septal myectomy and the modified Konno procedure, demonstrate efficacy in medication-refractory cases but carry higher complication risks in younger patients. The critical research gap lies in developing targeted therapeutic approaches for pediatric-specific HCM subtypes, particularly those associated with syndromic and metabolic disorders. Additionally, risk stratification models for sudden cardiac death prevention remain inadequately validated in pediatric populations. This review identifies the urgent need for pediatric-focused clinical trials investigating both conventional and novel therapies alongside the development of age-appropriate risk assessment tools to guide personalized management strategies for this vulnerable population.

05 May 2025·JOURNAL OF GENERAL PHYSIOLOGY

Myosin binding protein-C modulates loaded sarcomere shortening in rodent permeabilized cardiac myocytes

Article

Author: Davis, Daniel J. ; Hanft, Laurin M. ; Kalogeris, Theodore J. ; McDonald, Kerry S. ; Veteto, Adam B.

During the ejection phase of the cardiac cycle, left ventricular (LV) cardiac myocytes undergo loaded shortening and generate power. However, few studies have measured sarcomere shortening during loaded contractions. Here, we simultaneously monitored muscle length (ML) and sarcomere length (SL) during isotonic contractions in rodent permeabilized LV cardiac myocyte preparations. In permeabilized cardiac myocyte preparations from rats, we found that ML and SL traces were closely matched, as SL velocities were within ∼77% of ML velocities during half-maximal Ca2+ activations. We next tested whether cardiac myosin binding protein-C (cMyBP-C) regulates loaded shortening and power output by modulating cross-bridge availability. We characterized force–velocity and power–load relationships in wildtype (WT) and cMyBP-C deficient (Mybpc3−/−) mouse permeabilized cardiac myocyte preparations, at both the ML and SL level, before and after treatment with the small molecule myosin inhibitor, mavacamten. We found that SL traces closely matched ML traces in both WT and Mybpc3−/− cardiac myocytes. However, Mybpc3−/− cardiac myocytes exhibited disproportionately high sarcomere shortening velocities at high loads. Interestingly, in Mybpc3−/− cardiac myocytes, 0.5 µM mavacamten slowed SL-loaded shortening across the force–velocity curve and normalized SL shortening velocity at high loads. Overall, these results suggest that cMyBP-C moderates sarcomere-loaded shortening, especially at high loads, at least in part, by modulating cross-bridge availability.

01 May 2025·AMERICAN JOURNAL OF CARDIOLOGY

Real-World Impact of Mavacamten on Diastolic Function in Hypertrophic Cardiomyopathy: Insights Beyond Gradient Reduction

Author: Rao, Roopa A ; Das, Mithilesh K

186

News (Medical) associated with Mavacamten

15 Apr 2025

·pipelinereview.com

Bristol Myers Squibb Provides Update on Phase 3 ODYSSEY-HCM Trial

PRINCETON, NJ, USA I April 14, 2025 I Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 ODYSSEY-HCM trial evaluating Camzyos (mavacamten) for the treatment of adult patients with symptomatic New York Heart Association (NYHA) class II-III non-obstructive hypertrophic cardiomyopathy (nHCM) did not meet its dual primary endpoints of changes from baseline to Week 48 compared to placebo in the Kansas City Cardiomyopathy Questionnaire – Clinical Summary Score (KCCQ-23 CSS) and peak oxygen consumption (pVO2). No new safety signals were observed. “The ODYSSEY-HCM trial, the largest and longest-duration study completed to date in patients with non-obstructive HCM, tested the hypothesis of whether a cardiac myosin inhibitor would improve measures of feel and function for these patients, showing clinical benefits similar to what we have seen in obstructive HCM,” said Milind Desai, MD, MBA, Vice Chair in the Heart, Vascular & Thoracic Institute and Director of the HCM Center, Cleveland Clinic. “The findings of this trial help us understand that obstructive HCM and non-obstructive HCM are two unique diseases. Through long-term trials and real-world data from thousands of patients with symptomatic obstructive HCM, we have seen the meaningful impact that Camzyos has on improving the quality of life for patients living with this condition. ODYSSEY-HCM indicates that we must consider new ways of thinking about potential treatment approaches for non-obstructive HCM. We want to thank the patients and investigators for their efforts in completing this important trial and their commitment to advancing the scientific understanding of this complex disease.” “While these results are disappointing, the ODYSSEY-HCM trial meaningfully contributes to the understanding of non-obstructive HCM, a disease where there remains a significant need for new treatment options,” said Roland Chen, MD , senior vice president, drug development, Immunology and Cardiovascular Medicines, Bristol Myers Squibb. “These findings represent the first Phase 3 clinical trial data for a cardiac myosin inhibitor in non-obstructive HCM. Importantly, these results do not change the favorable benefit-risk profile that has been consistently demonstrated across our Camzyos clinical trials in obstructive HCM and the robust body of real-world effectiveness and safety evidence showing its benefit for people living with obstructive HCM around the world.” Bristol Myers Squibb will work with key investigators to share detailed results with the scientific community in the future. Bristol Myers Squibb thanks the patients and investigators who participated in the ODYSSEY-HCM clinical trial. About ODYSSEY-HCM ODYSSEY-HCM ( NCT05582395 ) is a Phase 3 randomized, double-blind, placebo-controlled trial that enrolled 580 adult patients with symptomatic (NYHA class II or III) non-obstructive hypertrophic cardiomyopathy (nHCM) worldwide. The dual-primary endpoints for the trial were to examine changes from baseline in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS) and peak oxygen consumption (pVO2) at Week 48. Secondary endpoints included change from baseline in ventilatory efficiency (VE/VCO2), NYHA functional class, N-terminal pro B-type natriuretic peptide (NT-proBNP) levels, and the Hypertrophic Cardiomyopathy Symptom Questionnaire-Shortness of Breath (HCMSQ-SoB) at Week 48. About CAMZYOS ® (mavacamten) CAMZYOS ® (mavacamten) is the first and only cardiac myosin inhibitor approved in the U.S., indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms, and in the European Union, indicated for the treatment of symptomatic (NYHA, class II-III) oHCM in adult patients. It has also received regulatory approvals in more than 50 countries and regions across five continents. CAMZYOS is a selective, reversible, allosteric inhibitor of cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In oHCM patients, myosin inhibition with CAMZYOS reduces dynamic left ventricular outflow tract (LVOT) obstruction and improves cardiac filling pressures. These effects on the heart translate to improvement in symptoms and ability to be active in symptomatic patients with oHCM. CAMZYOS U.S. INDICATION CAMZYOS ® (mavacamten) is a cardiac myosin inhibitor indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. Please see U.S. Full Prescribing Information , including Boxed WARNING and Medication Guide . About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , X , YouTube , Facebook and Instagram . SOURCE: Bristol Myers Squibb

Phase 3AHAClinical ResultDrug Approval

15 Apr 2025

·www.biopharmadive.com

Bristol Myers stumbles in bid to widen heart drug’s use

Dive Brief:Bristol Myers Squibb on Monday said its drug Camzyos failed a Phase 3 trial in people with a progressive heart condition, closing off an opportunity to expand use of a medicine it sees as a future blockbuster.According to Bristol Myers, Camzyos missed the dual main goals of a study focused on the non-obstructive form of HCM, or hypertrophic cardiomyopathy. It failed to meaningfully improve peak oxygen consumption as well as scores on an assessment of heart health. The company didnt provide study details, but said more information will be shared with the scientific community in the future.Camzyos was acquired through the $13 billion buyout of MyoKardia in 2020 and two years later became the first drug cleared for use in the obstructive and more common form of the disease. Biotechnology companies Cytokinetics and Edgewise Therapeutics are developing similar medicines that are both in the advanced stages of clinical testing. Cytokinetics drug, aficamten, could be approved in the U.S. later this year.Dive Insight:Camzyos setback carries implications for Bristol Myers as well as the companies trying to develop better medicines for HCM, a chronic condition characterized by a potentially deadly stiffening of the heart muscle.Bristol Myers is bracing for the losses of billions of dollars in yearly revenue when the patents expire for its cancer immunotherapy Opdivo and blood thinner Eliquis. The company already announced a cost-cutting plan under CEO Chris Boerner, and is looking to a handful of medicines, Camzyos among them, to grow sales in the years ahead.Camzyos generated $602 million in sales in 2024. But Bristol Myers has said that number could reach $4 billion in 2029, and one way to get there was a clearance in the non-obstructive setting, which is estimated to account for about a third of the people with the condition. The setback is a disappointment, wrote Leerink Partners analyst David Risinger, though Bristol Myers could rebound if a series of coming Phase 3 readouts are successful.The study failure also stirred debate among analysts as to whether drugs like Camzyos, known as cardiac myosin inhibitors, can help all people with HCM. Camzyos has proven beneficial for the obstructive form that impedes blood flow and can lead to heart failure as well as other potentially serious complications. The non-obstructive type doesnt, but can still put people at risk of poor health outcomes. The new study tested whether Camzyos might be similarly helpful for those people as well.The fact that Camzyos wasnt suggests obstructive and non-obstructive HCM are two unique diseases, said Milind Desai, the director of the Cleveland Clinics HCM Center, in a statement provided by Bristol Myers.Analysts were mixed on whether the cardiac myosin inhibitors Cytokinetics and Edgewise are developing might similarly struggle in non-obstructive HCM. Cytokinetics drug, aficamten, is under Food and Drug Administration review in obstructive HCM and in a Phase 3 trial in non-obstructive disease. Edgewises EDG-7500 could join it in late-stage testing next year.Matt Phipps, of the investment bank William Blair, wrote that Camzyos shaky performance in the latest study as well as Phase 2 testing in non-obstructive HCM patients may be more attributed to disease biology, which has negative implications for the whole drug class.That form of the disease is also more heterogeneous, making it tougher for a drug to succeed in testing, added RBC Capital Markets Leonid Timashev.Still, there are key differences between the drugs and their development plans that leave some analysts optimistic about Cytokinetics and Edgewises chances. Aficamten appeared to perform better in earlier testing in non-obstructive patients, and Cytokinetics Phase 3 study is designed differently, wrote Mizuho Securities Salim Syed. RBCs Timashev added that EDG-7500s unique mechanism could overcome the limitations of other medicines in non-obstructive HCM.We now see a 2-handed market split between Cytokinetics and Edgewise, Timashev wrote. '

Drug ApprovalPhase 3AcquisitionPhase 2Immunotherapy

15 Apr 2025

·www.biospace.com

BMS’ Camzyos Fails To Show New Cardiomyopathy Benefit in Phase III

iStock, apfDesign According to analysts at BMO Capital Markets, non-obstructive hypertrophic cardiomyopathy would have meant a $1.3 billion label expansion opportunity for Camzyos. Bristol Myers Squibb ’s myosin blocker Camzyos failed to ease clinical burden and improve oxygen consumption in patients with non-obstructive hypertrophic cardiomyopathy, the pharma announced on Monday. “This trial failure removes the opportunity for Camzyos in the 1/3 HCM patients who have the non-obstructive form of the disease,” analysts at BMO Capital Markets told investors in a Monday note. The late-stage stumble makes Camzyos’ label expansion into non-obstructive HCM “highly unlikely,” the analysts said. BMS did not present specific data in the news release, only revealing that Camzyos “did not meet its dual primary endpoints” in the Phase III ODYSSEY-HCM study, including 48-week improvement in peak oxygen consumption and other symptoms, including shortness of breath and blood markers for potential heart failure. In terms of safety, BMS did not document any new signals of concern. The pharma has promised to share detailed results from ODYSSEY-HCM “in the future,” as per its Monday release. Hypertrophic cardiomyopathy is a genetic disorder that causes heart muscle thickening. Depending on whether that thickening reduces flow of blood from the heart, the disorder can be classified as obstructive or non-obstructive. For BMO, the failure did not come as a big surprise: “We’ve always framed this trial as more challenging.” The analysts pointed to the difficulties of demonstrating strong and significant treatment benefits in patients with the less severe, non-obstructive form of the disease. Camzyos is an orally available cardiac myosin inhibitor that works by protein actions in muscular activation in the heart, with the goal of relaxing heart muscle and allowing the heart to fill with more blood. BMS gained ownership of Camzyos when it bought MyoKardia for $13.1 billion in October 2020. Two years later, the pharma secured the FDA’s approval for the drug, allowing its use in obstructive HCM. According to the BMO note, ODYSSEY-HCM will not change Camzyos’ opportunity in obstructive HCM, retaining a roughly $2.7 billion sales potential at peak. Still, the late-stage failure is a blow to BMS’ cardio portfolio, which will now miss out on more an additional 33,000 patients with the non-obstructive form of the disease—approximately $1.3 billion in earnings. BMO called this missed opportunity “meaningful.” BMS in the meantime is continuing to beef up its pipeline, with multiple “impactful catalysts” still lined up for 2025, according to BMO. “Top of mind for us remain the ARISE data evaluating the use of Cobenfy in adjunctive schizophrenia,” the analysts wrote, adding that this readout will come “any day now.” BMO is also looking forward to Cobenfy data in Alzheimer’s psychosis, expected in the latter half of 2025.

Drug ApprovalAcquisitionPhase 3

100 Deals associated with Mavacamten

External Link

KEGG	Wiki	ATC	Drug Bank
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R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hypertrophic obstructive cardiomyopathy	United States	Bristol Myers Squibb Co.	28 Apr 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	United States	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	China	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Japan	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Australia	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Austria	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Belgium	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Brazil	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Canada	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Czechia	Bristol Myers Squibb Co.	14 Dec 2022
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Denmark	Bristol Myers Squibb Co.	14 Dec 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05582395 (ODYSSEY-HCM, Company_Website) Manual	Phase 3	Hypertrophic Cardiomyopathy without Obstruction	580	Camzyos	nibgyeiolx(drmvlhvcvo) = did not meet eesrqggbsz (vyganuroxm ) Not Met View more	Negative	14 Apr 2025
				Placebo
NCT04766892 (EMBARK-HFpEF, CTgov)	Phase 2	Heart failure with normal ejection fraction	30	Mavacamten	phektyxtef = nleyjjdrsp xlmckqtskf (bsnjkwvebu, dlychuinhy - jqbdobmwjj) View more	-	20 Mar 2025
NCT03496168 (PIONEER-OLE, CTgov)	Phase 2	Hypertrophic obstructive cardiomyopathy	13	Mavacamtem (Mavacamtem)	jxhkqvwmto = ntlatypsbu kvkvbserfn (ivjgigmcql, sjxhcdihjx - enhfjfuewi) View more	-	09 Jan 2025
				Mavacamten (Mavacamten)	gnnukwmncv(kyohqiedqj) = tzgjtrgozn mtnudqiiyf (toyxmtbwmh, hwdvdhjbqo - izcqvhyajm) View more
NCT05414175 (HORIZON-HCM, CTgov)	Phase 3	Hypertrophic obstructive cardiomyopathy	38	Mavacamten	qavpgqtkld(lbrjrhrmcq) = puhsnausij dytxuvurfg (kdqwcqbcub, 31.55674) View more	-	04 Dec 2024
NCT03470545 (EXPLORER-HCM, Pubmed \| J Card Fail)	Phase 3	Hypertrophic obstructive cardiomyopathy	-	mavacamten (Age ≤60 years)	gmfavrewux(yrvusqetih) = hllzomaudv btnlnegyus (uzswlzpjzp ) View more	Positive	01 Dec 2024
				mavacamten (Age >60 years)	gmfavrewux(yrvusqetih) = wdyrcbnedx btnlnegyus (uzswlzpjzp ) View more
NCT05174416 (CTgov)	Phase 3	Hypertrophic obstructive cardiomyopathy	81	Mavacamten (Mavacamten)	wpognnusyw(evjyyygzla) = lqwhagdxau adlwwdedto (frlbwufnzu, 6.150) View more	-	19 Sep 2024
				Placebo (Placebo)	wpognnusyw(evjyyygzla) = heamkbqxkb adlwwdedto (frlbwufnzu, 8.535) View more
NCT03723655 (EXPLORER-LTE, BusinessWire) Manual	Phase 3	Hypertrophic obstructive cardiomyopathy	251	CAMZYOS® (mavacamten)	zmrckgjqhh(ghwgggbjli) = lvfoaawoty fleoewsjas (bttfufqtfm ) View more	Positive	01 Sep 2024
NCT04349072 (Pubmed \| JACC Cardiovasc Imaging)	Phase 3	Hypertrophic obstructive cardiomyopathy	-	Mavacamten	zkexbjbofa(vngxprsmfo) = aumdnffsmj cgxrhctlyr (lhrsayrmxs ) View more	-	01 Sep 2024
				Placebo	zkexbjbofa(vngxprsmfo) = lvnixulkcq cgxrhctlyr (lhrsayrmxs ) View more
ESC2024	Not Applicable	Hypertrophic obstructive cardiomyopathy	-	Mavacamten 2.5 mg	duyadmonrk(jnwsmtdmhe) = hgtcqkovxy jfqtajszjd (hlsuodhdnv ) View more	-	31 Aug 2024
				Mavacamten 5 mg	duyadmonrk(jnwsmtdmhe) = xgjkfgnviw jfqtajszjd (hlsuodhdnv ) View more
NCT05135871 (CTgov)	Phase 1	Cardiomyopathy, Hypertrophic	45	Mavacamten (Cohort 1: Mavacamten 15 mg)	obrwzjvsip(funzpwhaan) = kzkicynwom mdojghswbe (nkznqrldlr, 35.61) View more	-	25 Jul 2024
				Mavacamten (Cohort 2: Mavacamten 25 mg)	obrwzjvsip(funzpwhaan) = asucewjjmj mdojghswbe (nkznqrldlr, 50.49) View more

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