A Bristol Myers Squibb drug that the pharmaceutical giant added to its pipeline via a multi-billion dollar acquisition now has FDA approval, a decision that makes it the first authorized treatment for a rare inherited heart disorder that can become fatal.
For BMS, late Thursday approval of the drug, mavacamten, is an important win that brings the company another product to help offset looming patent expirations on key products. For patients, the regulatory decision introduces a treatment option for obstructive hypertrophic cardiomyopathy (oHCM), a condition that makes it harder for the heart to pump blood. The new BMS product, which will be marketed under the name “Camzyos,” is designed to target the source of this disorder.
Obstructive HCM stems from mutations to the gene that produces the heart muscle protein myosin. These mutated proteins lead to excessive contractions that cause the heart muscle to thicken. This thicker muscle makes it harder for the organ to pump blood and also obstructs the flow of blood to the rest of the body. Patients experience dizziness, fatigue, and shortness of breath. The condition can progress to heart failure. Current treatments for oHCM include beta blockers, drugs that cause the heart to beat more slowly, as well as calcium channel blockers that lower blood pressure. Camzyos is a small molecule designed to block the excess myosin. The drug is formulated as a capsule that patients take once daily.
The FDA based its decision on the results of a placebo-controlled Phase 3 study enrolling 251 patients. At 30 weeks, 37% of those treated with Camzyos showed improvement according to a measure of exercise capacity and symptoms of the disease compared with 17% in the placebo group.
While Camzyos is intended to head off heart failure stemming from oHCM, the way that the drug works can also contribute to heart failure. The drug has the effect of reducing left ventricular ejection fraction, which is the volume of blood the left ventricle pumps with each contraction. This reduced volume can lead to heart failure. Camyzos’s label carries a black box warning flagging this heart failure risk. The FDA said patients prescribed the drug must be monitored with echocardiograms to assess how the heart is working. Because of the heart failure risk, the drug is available only under a program that informs clinicians and patients about the dangers of the drug.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In a research note posted Friday, William Blair analyst Matt Phipps said that the black box warning was expected. He added that the drug’s approval comes at a key time. Revlimid, a blockbuster multiple myeloma that came to BMS with the 2019 Celgene acquisition, faces patent expirations starting this year. Phipps noted that generic competition has already begun to erode the drug’s sales.
Approval of Camzyos marks a payoff for BMS, which paid $13.1 billion in 2020 to acquire the drug’s developer, MyoKardia. Camzyos is the second first-in-class drug approval for BMS this year, coming five weeks after the FDA greenlighted cancer immunotherapy Opdualag. Both drugs enter give BMS additional products that can offset the coming sales declines for Revlimid.
Camzyos joins a BMS cardiovascular drug lineup anchored by anticoagulant Eliquis, a drug that accounted for $10.7 billion in 2021 sales, a 17.3% increase over the prior year. In first quarter 2022 financial results reported Friday, BMS said that Eliquis generated $3.2 billion in revenue, an 11% increase compared to the same period last year.
BMS set an $89,500 annual list price for Camzyos, which tops many of the estimates industry watchers set for the drug. It’s also well above the $12,000 to $15,000 annual price range that drug price watchdog group the Institute for Clinical and Economic Review said would be cost effective for the benefit provided by the medicine.
Speaking on a conference call Friday, BMS CEO Giovanni Caforio disagreed with ICER’s conclusion, saying that he did not think the group’s assessment was scientifically accurate and based on sound methodology. Chris Boerner, executive vice president and chief commercialization officer of BMS, said that as the first drug that targets the source of oHCM, there are no products comparable to Camzyos. He added that BMS thinks that the price reflects the value of the drug.
Image: Magicmine, Getty Images
