Aficamten

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 medicines 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 hearts 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.Myqorzos prescribing information includes somewhat less restrictive risk mitigation measures than whats mandated for people taking Bristol Myers Squibbs Camzyos, the only other available medication for the disease. Wall Street analysts and investors were closely watching whether Myqorzos risk management protocol might be different than Camzyos, as more permissive labeling could help Cytokinetics win market share.Dive Insight:Fridays 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 Myqorzos 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 hadnt seen in years.The investor interest stems from Myqorzos 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 Fridays approval, as the FDA decision and drug labeling met, rather than exceeded, investor expectations, wrote Stifels James Condulis. Theres still debate about the extent of Myqorzos differentiation, he added.According to Condulis, investors focus has already shifted to Cytokinetics next chance to clearly separate Myqorzo from Camzyos. Next year, itll 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. '

Cytokinetics has finally achieved its long-awaited goal of getting its first US approval. The company’s drug for a heart disease was approved in the US on Friday under the brand name Myqorzo. The pill, known generically as aficamten, may now be used to treat patients with a heart muscle thickening disorder called obstructive hypertrophic cardiomyopathy (oHCM), in which patients struggle to pump blood. The label granted by the FDA could give Myqorzo an advantage over the incumbent in this space, Bristol Myers Squibb’s Camzyos. A Cytokinetics spokesperson told Endpoints News on Friday that the company expects pricing “to be in line with current CMI therapy.” One crucial aspect is the drugs’ respective risk evaluation and mitigation strategies (REMS) — the patient safety provisions that must be followed when the drugs are dispensed. Both drugs inhibit production of a heart muscle protein called cardiac myosin, and as such carry a risk of causing heart failure. Cytokinetics initially didn’t submit a REMS for Myqorzo. The FDA later requested one, and extended the decision date by three months. Myqorzo’s label warns against giving the drug to patients whose left ventricular ejection fraction — a measure of how well the heart pumps blood — is less than 55%. It says to decrease the dose if LVEF is under 50% and equal to or above 40%. Myqorzo should be interrupted if a patient’s LVEF is under 40% or if they are experiencing heart failure symptoms. Once a patient is on their maintenance dose, echocardiograms will be required every six months for patients with LVEF over 55%, and every three months if LVEF is over 50% but under 55%. Camzyos’ label also recommends not giving the drug to patients whose LVEF is less than 55%. But if a patient’s LVEF drops below 50%, they must stop taking the drug, wait a month, and then restart at a lower dose. Echocardiograms are required every six months for patients on the drug. Myqorzo’s label also has no requirement for monitoring of drug-drug interactions. Whereas for Camzyos , dose reduction and additional monitoring is required for patients taking CYP2C19 inhibitors. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology,” Cytokinetics CEO Robert Blum said in a statement. “I’m pleased that the approved label and REMS reflect the distinct characteristics of Myqorzo including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile,” he added. Myqorzo is expected to be available in the US in January. The drug was also approved as Myqorzo in China on Wednesday and is expected to gain approval in the EU in the first quarter of 2026 under the same name.