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Blueprint wins key FDA approval for rare disease drug

May 23, 2023

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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.

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