Avapritinib is under clinical development by Blueprint Medicines and currently in Phase II for Refractory Acute Myeloid Leukemia. According to GlobalData, Phase II drugs for Refractory Acute Myeloid Leukemia have a 21% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Avapritinib’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

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 relapsed/refractory acute myeloid leukemia (AML), 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 is a precision therapy company that invented medicines for the treatment of cancer and blood disorders. The company product pipeline includes AYVAKIT (avapritinib) medicine to treat gastrointestinal stromal tumor (GIST) and GAVRETO (pralsetinib) for non-small cell lung cancer in adults, BLU-263 targeting indolent systemic mastocytosis. Blueprint Medicines also offers GAVRETO, BLU-701, BLU-945, BLU-451, BLU-222 and BLU-852. The company 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 discovery which combines bioinformatics and drug design capabilities to develop its medicines. Blueprint Medicines is headquartered in Cambridge, Massachusetts, the US.

For a complete picture of Avapritinib’s drug-specific PTSR and LoA scores, buy the report here.

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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 clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.