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FDA Approves New Treatment for Rare Blood Disorder

Rye Brook, N.Y., June 6, 2023 – The U.S. Food and Drug Administration (FDA) recently approved avapritinib (Ayvakit®) to treat adults with indolent systemic mastocytosis. 

Systemic mastocytosis occurs when the body produces irregular mast cells, which are a type of white blood cell. 

Symptoms of indolent (slow growing) systemic mastocytosis often go unnoticed for years and may develop in an individual’s skin, liver, spleen, or gastrointestinal tract. Although it begins as a slow growing condition, indolent systemic mastocytosis can become cancerous in up to 3 in 10 adults who have it.

“The development of avapritinib, a treatment that targets the KIT gene, can be traced back to the tireless work and investments LLS made in imatinib, a related and groundbreaking targeted treatment known as Gleevec,” said Lee Greenberger, PhD, Chief Scientific Officer of LLS. “It is wonderful to see LLS research investments continue to pay off, this time bringing the first-ever disease-modifying treatment to patients with indolent systemic mastocytosis.”

The FDA based its decision to approve this new use of avapritinib, which was previously approved for an advanced form of the disease, on study results that showed this treatment plus supportive care is effective in improving disease symptoms. Patients with systemic mastocytosis often experience debilitating symptoms resulting in poor quality of life. 

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