Cytokinetics is awaiting a decision from the U.S. Food and Drug Administration (FDA) regarding its drug aficamten, intended for the treatment of obstructive hypertrophic cardiomyopathy (oHCM). The FDA action date is set for December 26, and if approved, this would be Cytokinetics’ first drug to receive approval in the United States under its own name after nearly three decades in business.
The company’s main competitor in this area is Bristol Myers Squibb’s (BMS) Camzyos, which was developed with early involvement from Cytokinetics before being spun off to MyoKardia. BMS acquired MyoKardia in 2020 for $13.1 billion and brought Camzyos to market in 2022. According to Cytokinetics CEO Robert Blum, “When it first acquired MyoKardia, BMS said Camyzos could earn $4 billion in sales at its peak. And they’re on their way.” In 2024 alone, Camzyos generated over $600 million in revenue for BMS.
Blum also indicated that aficamten could reach similar or even higher sales figures if it captures a significant share of the market. “If aficamten also captures a preferential share of the HCM market, analysts say it could bring in $4 billion at its peak as well,” he said, noting that total sales might reach up to $10 billion with further product expansion.
“Twenty-seven years ago, we embarked on this journey of developing inhibitors of cardiac disease,” Blum stated. “Commercialization has been our plan since day one.”
Both drugs are cardiac myosin inhibitors designed to address oHCM by blocking myosin protein activity inside the heart muscle. Fady Malik, executive vice president of research and development at Cytokinetics, explained: “Think about the heart ejecting blood like a garden hose.” In patients with oHCM, blood flow becomes restricted due to thickened heart tissue, leading to increased pressure and symptoms such as shortness of breath.
Current treatments include beta blockers—often ineffective—and surgical procedures such as myectomy or even heart transplantation for advanced cases.
Malik described several drawbacks associated with Camzyos: slow dose titration requiring monthly checks during initial treatment months and potential risks if dosing needs adjustment. He contrasted this with aficamten’s faster titration schedule: “Aficamten…can be titrated every two weeks,” enabling more flexible adjustments.
Data released by Cytokinetics from the Phase III MAPLE-HCM trial showed improved exercise capacity among patients taking aficamten compared to those receiving standard care beta blockers. Analysts at Truist Securities called these results potentially “best-in-class” and supportive of using aficamten as a first-line therapy.
On December 17th, Cytokinetics announced that aficamten had received regulatory approval for oHCM in China—a milestone triggering a $7.5 million payment from Sanofi, which holds rights to commercialize the drug there.
The global market for oHCM is estimated at around $1 billion annually according to Blum; however, Cytokinetics aims to expand into non-obstructive HCM indications as well through ongoing studies such as ACAIA-HCM. The company reported positive results from a Phase II trial for non-obstructive HCM earlier this year.
Some analysts remain cautious about future revenue projections; following Chinese approval news on Wednesday, Truist modeled peak revenues at $3.4 billion for oHCM use and an additional adjusted peak revenue of $460 million from non-obstructive applications.
Cytokinetics plans independent commercialization efforts in the U.S. and Europe but may consider partnerships elsewhere globally. Addressing acquisition rumors, Blum said: “We thought we had agreed upon a deal with another party on being purchased, but that party walked away. We went back to plan A.” He added that should new opportunities arise they will act "to do the right thing for shareholder value."
Correction (Dec. 19): An earlier version misstated when data on non-obstructive HCM was released; it was published in March 2023 rather than September.
