Advances in Treatment of Obstructive Hypertrophic Cardiomyopathy - Episode 13


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A panel of experts in cardiovascular medicine review the use of mavacamten as seen in the VALOR-HCM study for patients with symptomatic oHCM who are eligible for septal reduction therapy.

James Januzzi, MD: What about VALOR-HCM? I’m curious about this one.

Steve R. Ommen, MD: VALOR-HCM was the next-level study that was a direct response to the fact that in EXPLORER, most of the patients enrolled were NYHA [New York Heart Association] class II patients. For surgery and ablation, most patients are class III or higher, so we should compare those 2 groups. That’s what VALOR-HCM was designed to do. For patients who were referred for consideration of septal reduction therapy, let’s randomize them in a trial. That’s what was done in VALOR-HCM. The results of that are promising: 75% of the placebo patients went on to septal reduction therapy during the 16 weeks of the study vs only just under 20% of the patients with active drugs. It was a tremendously different outcome in a period of 16 weeks.

Martin S. Maron, MD: I’ll expand on that by saying that in the context of time, it was a short period of time.

James Januzzi, MD: Sixteen weeks is short. Especially with a here-and-gone set of symptoms.

Martin S. Maron, MD: There’s a limited amount we can take away from that in terms of how effective these drugs are in either delaying or eliminating the need for definitive invasive septal reduction therapy. It’s too short a period of time.

James Januzzi, MD: That’s very useful. Javed, let me ask you in general terms about approval of a drug. The FDA looks at it, there’s time to review, time to respond, and then there’s a date that’s given for when the drug is supposed to be approved. What do we know about mavacamten [Camzyos] at this point?

Javed Butler, MD, MPH, MBA: From the perspective of the efficacy and improvement in functional capacity and quality of life, those data, I don’t think that there’s any concern or challenge there. The issue here is all the discussion that we’re having right now, which is how to monitor this therapy in the long run. Those are the considerations, figuring out the long-term monitoring, how frequently echocardiography should be done, how frequently the post-approval study be done, and what it should look like. Those are the discussions where I can see that the regulatory agencies will take some time to come up with a plan where we make sure that the patients who need this therapy and deserve this therapy get it.

At the same time, we have enough safeguards that we learn, and that learning could be to what John is saying, that we should do this for a year and learn that we don’t need to do this or that we don’t need to do this as frequently or that we should do this differently. I assume that those things will come to a conclusion in the near future, but that’s what we’re awaiting.

James Januzzi, MD: We’re waiting to hear. Once regulatory approval is granted, assuming that it is, there will be follow-up.

Javed Butler, MD, MPH, MBA: Yes.

James Januzzi, MD: There’s a planned long-term registry to look at patients with HCM [hypertrophic cardiomyopathy] treated with and without mavacamten to understand a lot of the questions we’re talking about. That will be interesting to see.

Transcript Edited for Clarity