Avapritinib is a Small Molecule owned by Blueprint Medicines, and is involved in 23 clinical trials, of which 9 were completed, 13 are ongoing, and 1 is planned.

Avapritinib is a potent inhibitor of alpha-type platelet-derived growth factor receptor (PDGFRa) and KIT Exon 17 mutants. It also inhibits KIT D816V and D842V mutants. These are tyrosine kinase receptor mutations that are found in neoplastic mast cells. The drug candidate by inhibiting KIT mutants selectively treats the underlying cause. The PDGF receptors play essential and complementary roles in angiogenesis. There by the drug candidate exhibits antineoplastic effects by antagonizing the actions of PDGFRa.

The revenue for Avapritinib is expected to reach a total of $15.9bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Avapritinib NPV Report.

Avapritinib is originated and owned by Blueprint Medicines. CStone Pharmaceuticals is the other company associated in development or marketing of Avapritinib.

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.

The company reported revenues of (US Dollars) US$180.1 million for the fiscal year ended December 2021 (FY2021), a decrease of 77.3% over FY2020. The operating loss of the company was US$642 million in FY2021, compared to an operating profit of US$308.7 million in FY2020. The net loss of the company was US$644.1 million in FY2021, compared to a net profit of US$313.9 million in FY2020. The company reported revenues of US$66 million for the third quarter ended September 2022, an increase of 80.5% over the previous quarter.

Quick View – Avapritinib

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

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

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.