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2022-10-21| Trials & Approvals

Bristol Myers Squibb’ sNDA for Rare Heart Disease Treatment Accepted by the FDA

by Max Heirich
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The United States Food and Drug Administration (FDA) accepted Bristol Myers Squibbs’ supplemental new drug application (sNDA) for CAMZYOS® (mavacamten). The sNDA expands the the treatment for symptomatic obstructive hypertrophic cardiomyopathy (HCM), a rare heart disease, for the reduction of the need for septal reduction therapy. BMS’ based its submission on the results of the Phase 3 VALOR-HCM Trial.

Related Article: FDA Pushes Biogen’s ALS Drug Approval Date Back Three Months

Expanding a Rare Heart Disease Treatment’s Use

Currently, CAMZYOS is a myosin inhibitor approved for the treatment of the rare disease HCM. The main symptom of the disease is a thickening of the muscles of a patient’s heart; as a result, the heart struggles to pump blood as efficiently. This results in shortness of breath, chest pain, or changes in the heart’s electrical system, resulting in life-threatening irregular heart rhythms (arrhythmias) or sudden death.

In April of this year, the FDA approved CAMZYOS for the treatment of HCM. However, BMS sought the expansion of the treatment’s approval to reduce HCM patients’ need for septal reduction therapy. Used for obstructive HCM, this therapy aims for further treatment in patients with limiting symptoms, despite prior treatment. 

Detracting against septal reduction therapy, Roland Chen, MD, senior vice president and head of cardiovascular development, Global Drug Development at Bristol Myers Squibb, said, “Currently, it is recommended that many patients with severe symptomatic obstructive hypertrophic cardiomyopathy undergo SRT. This often requires either an open-heart surgical procedure or septal ablation procedure – both specialized care options.”

The Phase 3 VALOR-HCM Trial compared CAMZYOS to a placebo with the end goal of determining which treatment results in a lessened need for septal reduction therapy. Overall, CAMZYOS won out, meeting the trial’s primary end goal.

As a result of the trial’s result, BMS filed an sNDA for CAMZYOS, which the FDA accepted on October 21.

Chen went on to say on the acceptance, “FDA acceptance of the filing for this expanded indication has the potential to strengthen the profile of CAMZYOS, while further reinforcing our commitment to delivering transformative cardiovascular therapies to patients.”

The FDA assigned a target action date for the sNDA on June 16, 2023. Due to the drugs’ prior approval, as well as the results of the VALOR trial, it is safe to say that the FDA will likely issue a positive opinion. 

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