- Blueprint Medicines has won Food and Drug Administration approval to sell its cancer drug Ayvakit to treat a rare blood disorder called advanced systemic mastocytosis.
- The verdict offers a bigger sales opportunity for Blueprint, which has been methodically securing FDA approvals since its first in January 2020. Previously, Ayvakit was cleared for patients with ultra-rare gastrointestinal stromal tumors, or GISTs.
- Ayvakit is designed to address the underlying cause of advanced systemic mastocytosis, in which a type of white blood cell known as a mast cell proliferates and causes damage in the body. The condition is usually caused by a mutation in cells called KIT D816V, which Ayvakit works to block.
Analysts forecast Blueprint's drug will likely quickly outpace Novartis' Rydapt as an option for patients with advanced systemic mastocytosis. Studies of Ayvakit showed a higher response rate than those testing Rydapt, and some of the patients on Ayvakit had previously been treated with Rydapt.
"Ayvakit is a clearly better agent," Raymond James analyst Dane Leone wrote in a note to investors. Leone expects sales of $13 million this year, rising to more than $400 million in 2026. Analysts at SVB Leerink see the drug on a similar track, with peak sales of about $980 million in 2030.
Last year, Ayvakit generated $21 million in net revenue for Blueprint.
Ayvakit's new label eases some concern investors had, analysts said. There is no black-box warning about safety issues, and a restriction limiting the drug's use to patients with a certain threshold of platelets is in line with the parameters used in the company's studies, Leone wrote.
While other targeted cancer companies have been snapped up by large pharmaceutical companies — most notably, Loxo Oncology and Array BioPharma, which were respectively bought for $8 billion and $11 billion — Blueprint has remained independent. The biotech has still engaged in deals, though, such as a partnership with Roche focused on the cancer medicine Gavreto.
Roche and Blueprint won approval for Gavreto to treat tumors with abnormalities in a gene known as RET in September 2020. In December, they won FDA clearance for an expanded label for the drug. Between its initial approval and the end of last year, Gavreto generated almost $1 million in net revenue, according to Blueprint's most recent annual report.