The Bristol Myers Squibb drug, the pharma giant added to its pipeline in a multibillion-dollar acquisition, now has FDA Approvedmaking it the first decision to authorize a treatment for a potentially fatal rare genetic heart disease.
The drug mavacamten, approved late Thursday, was a major win for BMS, bringing another product to the company to help offset an impending patent expiry on a key product. For patients, the regulatory decision introduced a treatment option for obstructive hypertrophic cardiomyopathy (oHCM), a disease that makes it harder for the heart to pump. The new BMS product, which will be marketed under the name “Camzyos,” is designed to target the root cause of the disease.
Obstructive HCM results from mutations in the gene that produces the cardiac protein myosin. These mutated proteins cause excessive contraction, which can lead to thickening of the heart muscle. This thicker muscle makes it harder for the organ to pump blood and also blocks blood flow to the rest of the body. Patients experience dizziness, fatigue, and shortness of breath. This condition may progress to heart failure. Current treatments for oHCM include beta-blockers, drugs that make the heart beat more slowly, and calcium channel blockers that lower blood pressure. Camzyos is a small molecule designed to block excess myosin. The drug is formulated into capsules that patients take once a day.
The FDA’s decision was based on results from a placebo-controlled Phase 3 study that enrolled 251 patients. At 30 weeks, 37 percent of patients treated with Camzyos showed improvement, compared with 17 percent of the placebo group, as measured by exercise capacity and disease symptoms.
While Camzyos is designed to stop heart failure caused by oHCM, the way the drug works can also cause heart failure. The drug has the effect of lowering left ventricular ejection fraction, which is the amount of blood pumped by the left ventricle each time it contracts. This reduced volume can lead to heart failure. Camyzos’ label carries a boxed warning about this heart failure risk. The FDA said patients prescribed the drug must be monitored by echocardiography to assess how well the heart is working. Because of the risk of heart failure, this drug should only be used in a program that informs clinicians and patients of the drug’s dangers.
In a research note released on Friday, William Blair analyst Matt Phipps said the black box warning was to be expected. He added that the drug’s approval comes at a critical time. Revlimid, the blockbuster multiple myeloma BMS acquired Celgene in 2019, faces patent expirations starting this year. Phipps noted that generic competition has begun to eat into the drug’s sales.
Camzyos’ approval marks BMS buys drug developer MyoKardia for $13.1 billion in 2020. Camzyos is BMS’ second first-in-class drug approval this year, coming five weeks after approval FDA approves cancer immunotherapy OpdualagThe entry of both drugs provides BMS with additional products that could offset Revlimid’s upcoming sales decline.
BMS set an annual list price of $89,500 for Camzyos, which exceeds the estimates set by many industry watchers for the drug.also much higher than Price range of $12,000 to $15,000 per year The Institute for Clinical and Economic Review said the drug price watchdog group was cost-effective for the benefits the medicines provided.
Speaking on a conference call on Friday, BMS Chief Executive Giovanni Caforio disagreed with ICER’s conclusions, saying he believed the group’s assessment was scientifically accurate and not based on sound methodology. Chris Boerner, executive vice president and chief commercialization officer at BMS, said that as the first drug to target a source of oHCM, no product can match Camzyos. Bristol-Myers Squibb believes the price reflects the value of the drug, he added.
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