Avapritinib is under clinical development by Blueprint Medicines and currently in the Phase I and Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Avapritinib’s likelihood of approval (LoA) and phase transition for Solid Tumor took place on 05 Dec 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Avapritinib Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Avapritinib overview

Avapritinib (Ayvakit, Ayvakyt) is an anti-neoplastic agent. It is formulated as film coated tablets for oral route of administration. 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, indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation, is indicated for the treatment of 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 (MCL).

Avapritinib is under development for the treatment of metastatic gastrointestinal stromal tumor, metastatic melanoma, non-advanced systemic mastocytosis (SM), adenocarcinoma of the gastroesophageal junction, breast cancer, sarcomas, colorectal cancer, lung cancer, central nervous system (CNS) tumor, high-grade glioma, systemic mastocytosis associated with clonal hematological non-mast cell disorders (SM-AHNMD) and mast cell leukemia and treatment . It is a small molecule administered orally in the form of tablet and capsule. The drug candidate inhibits alpha-type platelet-derived growth factor receptor and KIT Exon 17 mutants including KIT D816V.

Blueprint Medicines overview

Blueprint Medicines, formerly Hoyle Pharmaceuticals, is precision therapy company that inventing medicines for people with cancer and blood disorders. The company product pipeline includes AYVAKIT (avapritinib) medicine to treat gastrointestinal stromal tumor (GIST) and GAVRETO (pralsetinib) against non-small cell lung cancer in adults, BLU-263 targeting indolent systemic mastocytosis. It also offer GAVRETO, BLU-701, BLU-945, BLU-451, BLU-222 and BLU-852. Blueprint Medicines is advancing pralsetinib inhibitor against RET (Ret Proto-Oncogene) modified medullary thyroid carcinoma and other solid tumors and avapritinib to treat advanced systemic mastocytosis. The company utilizes its discover which combines bioinformatics and drug design capabilities to develop its medicines. Blueprint Medicines is headquartered in Cambridge, Massachusetts, the US.

Quick View Avapritinib LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Avapritinib
Administration Pathway
  • Oral
Therapeutic Areas
  • Gastrointestinal
  • Oncology
Key Developers
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.