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Avapritinib

Generic Name
Avapritinib
Brand Names
Ayvakit, Ayvakyt
Drug Type
Small Molecule
Chemical Formula
C26H27FN10
CAS Number
1703793-34-3
Unique Ingredient Identifier
513P80B4YJ
Background

Avapritinib, or BLU-285, is a selective tyrosine kinase inhibitor of KIT and platelet derived growth factor receptor alpha indicated for the treatment of unresectable, metastatic gastrointestinal stromal tumors and advanced systemic mastocytosis. It is one of the first medications available for the treatment of multidrug resistant cancers. Avapritinib shares a similar mechanism with ripretinib.

Avapritinib was granted FDA approval on 9 January 2020 and EMA approval on 24 September 2020.

Indication

Avapritinib is indicated for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

It is also used to treat adult patients with advanced systemic mastocytosis (AdvSM). AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia. However, it is not recommended for the treatment of patients with AdvSM with platelet counts of less than 50 X 10 L.

Associated Conditions
Advanced Systemic Mastocytosis (AdvSM), Aggressive Systemic Mastocytosis, Indolent Systemic Mastocytosis, Mast Cell Leukemia (MCL), Metastatic Gastrointestinal Stromal Tumor (GIST), Systemic Mastocytosis With an Associated Hematological Neoplasm, Unresectable Gastrointestinal stromal tumor
Clinical Trials
Related News

Deciphera's Qinlock Fails Phase 3 Trial in Second-Line GIST, Shares Plummet 75%

• Deciphera Pharmaceuticals' Qinlock failed to outperform Pfizer's Sutent in second-line gastrointestinal stromal tumor treatment, with progression-free survival of 8.0 versus 8.3 months respectively. • The disappointing INTRIGUE trial results severely impact Qinlock's projected blockbuster potential, previously estimated at $1.5 billion in annual sales. • Currently approved as a fourth-line therapy, Qinlock generates modest quarterly sales of around $20 million, with a pending European approval offering limited upside potential.

Avapritinib Shows Promising Improvements in Bone Density for Advanced Systemic Mastocytosis Patients

Recent findings from the phase 2 PATHFINDER study reveal that avapritinib significantly improves bone density in patients with advanced systemic mastocytosis, offering new hope for managing this rare condition. The study highlights sustained improvements in lumbar T-scores and overall bone health, marking a significant advancement in treatment options.

Blueprint Medicines Announces Positive BLU-808 Phase 1 Data and 2025 Growth Strategy

• Blueprint Medicines projects a $4 billion peak revenue opportunity for its systemic mastocytosis (SM) franchise, driven by AYVAKIT's success and updated SM prevalence estimates. • Phase 1 trial results for BLU-808, an oral wild type KIT inhibitor, showed a wide therapeutic window and significant tryptase reductions, supporting its potential in allergic and inflammatory diseases. • The company anticipates achieving $2 billion in AYVAKIT revenue by 2030 and is initiating proof-of-concept studies for BLU-808 in various allergic and inflammatory conditions. • Blueprint Medicines has initiated a Phase 3 trial of elenestinib in indolent systemic mastocytosis (ISM) and is advancing CDK2 and CDK4 targeted protein degraders for breast cancer.

Avapritinib Demonstrates Long-Term Benefits in Advanced Systemic Mastocytosis

• Avapritinib (AYVAKIT) shows significant survival benefits in treatment-naïve patients with advanced systemic mastocytosis (SM) compared to midostaurin in real-world outcomes. • Data from the PATHFINDER trial indicates that avapritinib leads to sustained improvements in bone density for advanced SM patients with low bone mass. • Ultra-sensitive KIT testing identifies previously undetected KIT mutations in indolent systemic mastocytosis (ISM) patients, suggesting a higher disease prevalence. • A predictive model using machine learning distinguishes between advanced SM and ISM with high accuracy, validated by an independent dataset from Dana-Farber Cancer Institute.
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