- The Food and Drug Administration has cleared a drug from biotechnology company Blueprint Medicines for treating people with a slow-moving, or “indolent,” form of the rare disease systemic mastocytosis.
- The drug, sold as Ayvakit, was initially approved in June 2021 for more limited use in advanced disease. The new indication covers adults with indolent systemic mastocytosis who are believed to account for anywhere from 90% to 95% of the roughly 32,000 people in U.S. with the disease.
- Approval was based on a study that showed Ayvakit and supportive treatment meaningfully reduced symptoms compared to a placebo and additional care. Blueprint is pricing the drug at its current cost for advanced disease — about $445,000 per year at list price — executives said on a conference call Monday.
The launch of Ayvakit in indolent systemic mastocytosis, or ISM, is being closely watched by investors and analysts.
Blueprint brought two drugs to market in recent years — Ayvakit and the cancer drug Gavreto — and since expanded use of each. But neither are big sellers, and the company still isn’t making money.
The biotech recorded a net loss of about $558 million last year on $111 million in net drug sales. And though it held nearly $1 billion in cash on its balance sheet at the end of the last quarter, Blueprint’s “high burn rate has been an overhang,” wrote Stifel analyst Bradley Canino earlier this month.
A successful launch in ISM could help. There are no available medicines that can change the disease’s course, and in testing Blueprint’s drug reduced the frequency of a constellation of unpredictable and potentially severe symptoms, such as pain and brain fog.
On a conference call Monday afternoon, executives emphasized the “broad” label Ayvakit received from regulators, which includes all ISM patients, and estimated a peak sales opportunity of more than $1.5 billion. “We are confident in our ability to grow that over time,” said Chief Commercial Officer Philina Lee.
Still, some Wall Street analysts remain skeptical about the drug’s potential, citing the effect size observed in testing and its high price. SVB Securities analyst Andrew Berens wrote that the prescribing information included an “erosion” in efficacy from a magnitude “that some investors had already seen as marginal” compared to standard care. Berens also noted the label’s mention of hematomas and hemorrhages in some patients, which “have not been previously characterized” and could become more common with longer drug exposure.
The data included in the label, a roughly 5.7-point placebo-adjusted reduction on a test measuring symptoms after six months of treatment, leaves “clinical significance open to debate,” added Piper Sandler analyst Christopher Raymond.
“The uptake and trajectory of the ISM launch remain unclear to us,” Raymond wrote.
Blueprint could also face competition in the future from biotech Cogent Biosciences, which has a rival ISM medicine in Phase 2 testing.
Shares of the company climbed about 4%, to around $59 apiece, in early trading Tuesday.