FOR IMMEDIATE RELEASE:
AYVAKIT® (avapritinib) Now Available from Onco360 for the Treatment of Adult Patients with Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST) Harboring a Platelet-Derived Growth Factor Receptor Alpha (PDGFRA) Exon 18 Mutation, Including PDGFRA D842V, Advanced Systemic Mastocytosis (AdvSM), and Indolent Systemic Mastocytosis (ISM)
Louisville, KY — Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected by Blueprint Medicines to be a specialty pharmacy partner for AYVAKIT® (avapritinib), which is a kinase inhibitor for the treatment of three indications:
- Adult patients with unresectable or metastatic Gastrointestinal Stromal Tumor (GIST) harboring a Platelet-Derived
Growth Factor Receptor Alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V
- Adult patients with Advanced Systemic Mastocytosis (AdvSM)
- Adult patients with Indolent Systemic Mastocytosis (ISM)
“Onco360 is honored to partner with Blueprint Medicines and become a specialty pharmacy provider for patients treated with AYVAKIT,” said Benito Fernandez, Chief Commercial Officer at Onco360. “We are dedicated to supporting the highly specialized needs of patients battling unresectable or metastatic PDGFRA Gastrointestinal Stromal Tumor (GIST) as well as those battling Advanced and Indolent Systemic Mastocytosis across the United States.”
According to the American Cancer Society (ACS), it is estimated that 4,000-6,000 cases of GIST are diagnosed in the United States each year. This accounts for less than 1% of all gastrointestinal tumors. The five-year relative survival rate (RS) for all stages at diagnosis is 85%. Unfortunately, the five-year RS for patients initially diagnosed with metastatic GIST is only 52%. According to the National Comprehensive Cancer Network (NCCN) Guidelines for Gastrointestinal Stromal Tumors, 5-10% of GIST cases have a mutation in the gene that encodes the platelet-derived growth factor receptor alpha (PDGFRA) receptor tyrosine kinase, including PDGRFA D842V. 1, 2
AYVAKIT is commercialized by Blueprint Medicines. The FDA approval of AYVAKIT for use in unresectable or metastatic PDGFRA exon 18-driven GIST was based on the results of the NAVIGATOR (NCT02508532) trial. Key trial endpoints included the overall response rate, duration of response, and safety.
Systemic mastocytosis (SM) is a rare disorder which affects approximately 32,000 people in the United States.7 Advanced SM includes subtypes aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). The median overall survival is approximately 3.5 years in patients with ASM, approximately two years in patients with SM-AHN, and only about 2 months in patients with MCL.8
The FDA approval of AYVAKIT for use in Advanced Systemic Mastocytosis was based on the results of EXPLORER (NCT02561988) and PATHFINDER (NCT03580655), two multi-center, single-arm, open-label clinical trials. Key trial endpoints included the overall response rate, duration of response, and safety.
The FDA approval of AYVAKIT for use in Indolent Systemic Mastocytosis is supported by findings from the double-blind, placebo-controlled, phase 2 PIONEER trial (NCT03731260), in which the addition of AYVAKIT to best supportive care was compared to placebo plus best supportive care. Key trial endpoints included the overall response rate, duration of response, and safety.9,10
For full prescribing information, please visit: AYVAKIT Prescribing Information
1 Key Statistics for Gastrointestinal Stromal Tumors (cancer.org) Accessed 5/8/23.
2 Gastrointestinal Stromal Tumors (NCCN) Accessed 5/8/23.
3 Avapritinib in advanced PDGRFA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR) Accessed 5/8/23.
4 AYVAKIT Prescribing Information Accessed 5/8/23
5 Safety and efficacy of avapritinib in advanced systemic mastocytosis (EXPLORER) Accessed 5/8/23
6 Efficacy and safety of avapritinib in advanced systemic mastocytosis (PATHFINDER) Accessed 5/8/23
7 Mastocytosis: Statistics (cancer.net) Accessed 5/8/2023.
8 Lim KH, Tefferi A, Lasho TL, et al. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood. 2009;113(23):5727-5736.
9 FDA approves Ayvakit (avapritinib) as the first and only treatment for indolent systemic mastocytosis. News release. Blueprint Medicines Corporation. May 22, 2023. Accessed May 23, 2023. https://ir.blueprintmedicines.com/news-releases/news-release-details/fda-approves-ayvakitr-avapritinib-first-and-only-treatment.
10 Blueprint Medicines announces positive top-line results from PIONEER trial of Ayvakit (avapritinib) in patients with non-advanced mastocytosis achieving primary and all key secondary endpoints. News release. Blueprint Medicines Corporation. August 17, 2022. Accessed May 23, 2023.