Camzyos®▼(mavacamten) granted Marketing Authorisation in Great Britain for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM)

Camzyos^®▼(mavacamten) granted Marketing Authorisation in Great Britain for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM)

• Mavacamten is a first-in-class selective, allosteric and reversible cardiac myosin inhibitor for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients[1]
• HCM is the most common inherited heart condition, estimated to affect 1 in 500 people in the UK[2]

(Uxbridge, Middlesex 31^st July 2023) – Bristol Myers Squibb (BMS) today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation in Great Britain for mavacamten, a once-daily oral medicine for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients. The National Institute for Health and Care Excellence (NICE) published final draft guidance of mavacamten for routine NHS access in England and Wales on 2 June[3] and will publish its final guidance shortly after marketing authorisation.

Professor Perry Elliott, Professor of Cardiovascular Medicine at University College London (UCL) commented: “Obstructive hypertrophic cardiomyopathy (oHCM) can have a significant impact on the physical and mental well-being of people affected by the condition. Symptoms are currently managed using medicines first developed decades ago, but these are often ineffective leaving cardiac surgery as the only option for some patients. The authorisation of a brand new oral treatment that targets the underlying pathophysiology of oHCM may represent an important step change in the treatment of this disease."

HCM is the most common inherited heart condition, estimated to affect 1 in 500 people in the UK.² The most common form of HCM, called obstructive HCM (oHCM), affects approximately 70% of patients.^[4]

Scott Cooke, General Manager of Bristol Myers Squibb UK & Ireland said: “At Bristol Myers Squibb, we are proud to be able to build upon our heritage in cardiovascular medicines, with a new focus on obstructive hypertrophic cardiomyopathy, a myocardial disease with significant unmet need. This authorisation from the MHRA brings us one step closer to potentially making this new first-in-class cardiac myosin inhibitor available for eligible patients in the UK, where there is yet to be a licensed therapy that targets the underlying cause of the disease.”

The marketing authorisation in Great Britain is based on pivotal data from two Phase III trials, EXPLORER-HCM¹ and VALOR-HCM.[5] The EXPLORER-HCM trial which evaluated mavacamten in patients with symptomatic oHCM versus placebo enrolled 251 patients randomly assigned to mavacamten (n=123) or placebo (n=128).¹ The primary composite endpoint of the EXPLORER-HCM trial included measured change in symptoms (NYHA class) and exercise capacity (pVO₂).¹ In the EXPLORER-HCM trial, mavacamten demonstrated superiority in achieving the primary composite endpoints compared to placebo at 30 weeks; 37% (45/123) versus 17% (22/128).¹ The VALOR-HCM study evaluated mavacamten as an add-on treatment for 112 patients (mavacamten n=56, placebo n=56) receiving standard of care and eligible for septal reduction therapy (SRT) according to the 2011 American College of Cardiology (ACC) / American Heart Association (AHA) guidelines.⁵ The primary composite endpoint of the study included patient decision to proceed with SRT or patients who remain SRT eligible at Week 16.⁵ Mavacamten demonstrated superiority with fewer patients achieving the primary composite endpoint compared to placebo at 16 weeks; 18% (10/56) versus 77% (43/56).⁵

Notes to editors

About mavacamten

Mavacamten (pronounced mah-vah-cam-ten) is a first-in-class selective, allosteric and reversible cardiac myosin inhibitor.¹ In HCM patients, cardiac myosin inhibition with mavacamten normalises contractility, reduces dynamic left ventricular outflow tract (LVOT) obstruction, and improves cardiac filling pressures.[6] This results in a reduction of actin-myosin cross-bridge formation, thereby reducing excessive heart contractions and improving the efficiency of the heart.¹ Mavacamten is taken as a once-daily oral capsule^{(2.5 mg, 5 mg, 10 mg and 15 mg)}.¹

About access to mavacamten

Mavacamten is now licensed for use across the UK. This authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA) grants a licence in Great Britain, while under the Northern Ireland Protocol, the marketing authorisation granted by the European Commission (EC) on 26 June 2023 authorises use in Northern Ireland. The EC decision also applies to the Republic of Ireland.

The National Institute for Health and Care Excellence (NICE) published final draft guidance following its appraisal of mavacamten for routine NHS access to mavacamten in England and Wales on 2 June³ and will publish its final guidance shortly after marketing authorisation. A separate submission has been made to the Scottish Medicines Consortium (SMC) and to the National Centre for Pharmacoeconomics (NCPE) in the Republic of Ireland.

Bristol Myers Squibb is committed to ensuring eligible patients can access this treatment as quickly as possible via UK health services and will continue to work in collaboration with all relevant authorities to help achieve this.

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/gb.

References

[1] Olivotto I, et al. 2020. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 396(10253), pp.759-769.

[2] Cardiomyopathy UK. Hypertrophic cardiomyopathy factsheet. Available at: https://www.cardiomyopathy.org/sites/default/files/2022-02/Hypertrophic%20cardiomyopathy%20factsheet%20January%202022.pdf. Accessed July 2023.

[3] National Institute for Health and Care Excellence. Mavacamten for treating symptomatic obstructive hypertrophic cardiomyopathy [ID3928]. Available at https://www.nice.org.uk/guidance/indevelopment/gid-ta10824/documents. Accessed July 2023.

[4] Prinz C, et al. 2011. The Diagnosis and Treatment of Hypertrophic Cardiomyopathy. Dtsch Arztebl Int. 108(13): 209–215.

[5] Desai MY, et al. 2022. Myosin inhibition in patients with obstructive hypertrophic cardiomyopathy referred for septal reduction therapy. J Am Coll Cardiol. 80(2), pp.95-108.

[6] European Medicines Agency (EMA). Camzyos (mavacamten) Summary of Opinion (initial authorisation). Available at: https://www.ema.europa.eu/en/documents/smop-initial/chmp-summary-positive-opinion-camzyos_en.pdf Accessed July 2023.