Dive Brief:

• The Food and Drug Administration has approved a new treatment for an inherited form of heart failure in a decision that also gives the medicine’s maker, Cytokinetics, the first U.S. approval in its nearly three-decade history.
• The agency on Friday approved Cytokinetics’ Myqorzo for adults with obstructive hypertrophic cardiomyopathy, a potentially deadly condition that impairs the heart’s ability to pump blood. The drug has specifically been cleared to help improve functional capacity and symptoms in patients whose disease severity is categorized as “Class II” or Class III.” It will be available in the second half of January, according to the company.
• Myqorzo’s prescribing information includes somewhat less restrictive risk mitigation measures than what’s mandated for people taking Bristol Myers Squibb’s Camzyos, the only other available medication for the disease. Wall Street analysts and investors were closely watching whether Myqorzo’s’ risk management protocol might be different than Camzyos’, as more permissive labeling could help Cytokinetics win market share.

Dive Insight:

Friday’s approval represents a breakthrough for Cytokinetics, a biotech that was founded in 1997 and is just now bringing its first product to market.

The company suffered two big setbacks in 2023, when one drug it had been developing for ALS failed in late-stage testing and the FDA rejected another would-be therapy for cardiovascular disease. But Myqorzo’s progress enabled a rebound. Over the last couple years, the drug emerged as a viable threat to Camzyos, which is expected to top $1 billion in sales this year. Cytokinetics was rumored to be a buyout target along the way, lifting shares to levels the company hadn’t seen in years.

The investor interest stems from Myqorzo’s potential to distinguish itself from Camzyos. Both drugs are so-called cardiac myosin inhibitors, designed to essentially relax the heart muscles and, in doing so, improve organ function. Both are now approved for the “obstructive,” or more common form of HCM.

Camzyos, though, has a strict warning on its label for the risk of heart failure and an FDA-mandated plan to manage that risk. The plan involves close patient monitoring, screening for drug interactions and regular echocardiograms that could result in dose adjustments if necessary. Myqorzo was handed a similar heart failure warning, but its risk protocol includes more “flexible” dose adjusting, less frequent echocardiograms and “minimal” drug interaction monitoring, wrote RBC Capital Markets’ Leonid Timashev.

Those factors “should materially lower barriers to prescribing” instead of Camzyos, he wrote.

Still, Cytokinetics shares were little changed upon Friday’s approval, as the FDA decision and drug labeling met, rather than exceeded, investor expectations, wrote Stifel’s James Condulis. There’s still “debate” about the “extent” of Myqorzo’s differentiation, he added.

According to Condulis, investors’ focus has already shifted to Cytokinetics’ next chance to clearly separate Myqorzo from Camzyos. Next year, it’ll report results from a trial testing its drug in people with the “non-obstructive” form of HCM. Camzyos failed a Phase 3 study in that setting, stirring debate that other drugs like it might similarly struggle. However, some analysts have suggested that Myqorzo, which has a wider potential range of safe and therapeutic doses, might have a better chance of success.

“We're bullish and expect [Myqorzo] to become the best [and] only drug for the entire HCM spectrum,” Condulis wrote.

The drug will have a list price “in line with” Camzyos and be revealed ahead of its availability in January, according to a spokesperson. It was cleared in China early last week. Camzyos launched with a yearly wholesale acquisition cost of $89,500.