#Avapritinib
Thrilled to share our work with Mariella Filbin, Johannes Gojo and Carl Koschmann, now published in @cp-cancercell.bsky.social We present a translational approach identifying the PDGFRA inhibitor avapritinib as a novel targeted treatment for pHGG with PDGFRA alterations www.cell.com/cancer-cell/...
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...
www.cell.com
March 13, 2025 at 3:30 PM
Endlich gibt es ein Medikament für meine seltene Erkrankung! Da kommt zumindest ein Funke Hoffnung auf... ich bin gespannt, ob man mir das Medikament überhaupt anbietet. 🤔

mastozytose-info.de/ayvakyt-avap...
AYVAKYT® (Avapritinib) erhält die Zulassung der Europäischen Kommission als erste und einzige Beh...
AYVAKYT® (Avapritinib) erhält die Zulassung der Europäischen Kommission als erste und einzige Behandlung für indolente systemische Mastozytose
mastozytose-info.de
December 18, 2023 at 7:12 PM
📣Proud of this work in collaboration with analytical groups. We propose a methodology for controlling the quality of avapritinib drug just published in Scientific Reports. @dquimicauib.bsky.social @biblioteca.uib.cat
www.nature.com/articles/s41...
Sustainable high-throughput microwell spectrophotometric methods for avapritinib quality control via charge-transfer complexation - Scientific Reports
Scientific Reports - Sustainable high-throughput microwell spectrophotometric methods for avapritinib quality control via charge-transfer complexation
www.nature.com
May 23, 2026 at 8:34 AM
Dr. Aram Bidikian and Yale colleagues share a case report in Leukemia & Lymphoma @tandfonline.bsky.social on the reversal of systemic mastocytosis-induced liver #cirrhosis with #avapritinib treatment.
tandfonline.com/doi/full/10....
@yale-hematology.bsky.social @yalepathology.bsky.social
Reversal of systemic mastocytosis-induced liver cirrhosis with avapritinib treatment: case report
Published in Leukemia & Lymphoma (Ahead of Print, 2026)
tandfonline.com
September 9, 2026 at 12:04 PM
Very proud of this collaboration with I.A. Darwish where we developed two smartphone-assisted colorimetric microwell methods for sustainable and high throughput analysis of avapritinib tablets. Just published in Sus. Chem. Phar.
@dquimicauib.bsky.social @biblioteca.uib.cat
doi.org/10.1016/j.sc...
August 7, 2026 at 7:44 AM
Kicking off #ASH24 is Daniel DeAngelo, MD, PhD’s research on Avapritinib treatment that created a highly accurate tool to predict between advanced and less severe forms of Systemic Mastocytosis.
December 7, 2024 at 6:38 PM
Translational data indicate that the PDGFRA/KIT inhibitor avapritinib shows preclinical activity and effective blood-brain barrier penetration in PDGFRA-altered high-grade glioma (HGG). Early clinical observations in a small cohort (n=8) showed a radiographic response in 3/7 cases.
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...
www.cell.com
June 3, 2026 at 12:34 PM
Quite an interesting article on targeting PDGFRA altered High-Grade Glioma
#ISRVT25
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib: Cancer Cell www.cell.com/cancer-cell/...
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...
www.cell.com
March 17, 2025 at 2:38 PM
Hoffnung bei #Hirntumoren bei #Kindern: Forschungsteam der #MedUni #Wien entdeckt gezielte Therapie gegen aggressive #Gliome. Der #PDGFRA-Inhibitor #Avapritinib zeigt erste Erfolge – Tumore, die resistent gegen Bestrahlung waren, sprechen an. Vielversprechend! #Krebs
www.cell.com/cancer-cell/...
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...
www.cell.com
March 14, 2025 at 12:18 PM
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib

www.sciencedirect.com/science/arti...
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is crucial to tumorigenesis and frequently genomically altered in high-grade glioma (HGG). In a comprehensive dataset of pediatric HGG (n = 261…
www.sciencedirect.com
April 23, 2025 at 12:50 PM
Blueprint has one drug – Avaykyt (avapritinib) – which is approved in the US, Europe and elsewhere to treat gastrointestinal stromal tumours, as well as the rare immunological disease systemic mastocytosis
Sanofi to buy Blueprint Medicines for over $9 billion
Blueprint has one approved drug and enhances Sanofi's immunology portfolio
www.chemistryworld.com
June 4, 2025 at 2:55 PM
AI makes treatment effects measurable: University of Basel researchers and colleagues show how AI can quantify skin changes in mastocytosis – an important step for clinical trials.
AI helps to evaluate skin lesions in rare disease more accurately
AI detects skin lesions in mastocytosis and shows that the drug avapritinib significantly reduces skin changes.
www.unibas.ch
March 19, 2026 at 9:29 AM
New editorial: Sweden's TLV Grants Limited Reimbursement for Avapritinib (Ayvakyt) in GIST and Systemic Mastocytosi… https://www.mattheneus.com/editorial/tlv-avapritinib-ayvakyt-limited-reimbursement-gist-systemic-mastocytosis-2026-03-24 #HTA #NMA #MAIC #EvidenceSynthesis #Pharma
March 26, 2026 at 2:50 PM
4-Year Data Underscore Durable Efficacy With Avapritinib in Advanced Systemic Mastocytosis @ASH_hematology #oncology www.onclive.com/view/4-year-...
4-Year Data Underscore Durable Efficacy With Avapritinib in Advanced Systemic Mastocytosis | OncLive
Deepti Radia, MBBS, BSc, MRCPI, FRCPath, MSc Med Ed, discusses 4-year data from the PATHFINDER trial of avapritinib in advanced systemic mastocytosis.
www.onclive.com
February 12, 2026 at 10:49 PM
#Avapritinib produced durable survival, demonstrated deep molecular and organ responses, and sustained bone benefits as first-line therapy for advanced #SystemicMastocytosis (SM), supporting long-term disease modification across SM subtypes.

Read here: https://bit.ly/49XipaM

#RareDisease #MedSky
Long-Term Avapritinib Use Sustains Responses in Advanced SM
Four-year follow-up demonstrated durable survival, sustained responses, and disease control with first-line avapritinib in advanced SM.
bit.ly
January 19, 2026 at 6:10 PM
In patients with indolent #SystemicMastocytosis (SM), treatment with #Avapritinib has been associated with statistically significant, clinically meaningful improvements in dermatologic manifestations of the disorder. Findings from the #PIONEER trial (#NCT03731260)

Learn more: https://bit.ly/4iqRVS0
Avapritinib Linked to Sustained Improvements in Skin Manifestations of Indolent SM
Patients with indolent SM and dermatologic involvement treated with avapritinib saw marked reductions in skin symptoms, skin color, and skin lesions.
bit.ly
November 25, 2025 at 5:40 PM
May is Brain Cancer Awareness Month. New research from the lab of Mariella Filbin, MD, PhD, suggests that avapritinib, an FDA-approved drug used to treat GI tumors, could also treat high-grade gliomas.

Learn more.
Repurposed Cancer Drug Could Help Treat Brain Tumors
A common gastrointestinal cancer drug that crosses the blood-brain barrier showed promise in patients with aggressive high-grade gliomas.
bit.ly
May 25, 2025 at 4:00 PM
New hope for #BrainTumors in #Children: Research team from #MedUni #Vienna discovers targeted therapy for aggressive gliomas. The #PDGFRA inhibitor #Avapritinib shows initial success – tumors resistant to radiation are responding. Promising! #Cancer
www.cell.com/cancer-cell/...
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...
www.cell.com
March 14, 2025 at 12:19 PM