Drug Detail

Information about Iclusig

Generic Name
Brand Name (US)
Drug Type
Tyrosine Kinase Inhibitor
Approval Status
Approved for a non-GIST cancer
Overall Strategy
GIST cell based
Block related tumor signal paths
Drug Category
KIT/PDGFRA inhibitor+ VEGF inhibitor (TKI)

Ponatinib is an Bcr-Abl/KIT/VEGFR inhibitor manufactured by ARIAD. This drug is primarily being developed as a Bcr-abl inhibitor for CML. Unlike most bcr-abl inhibitors, it inhibits the T315I mutation that has been a significant cause of resistance in CML.
Phase II clinical trials are in progress for GIST (2013). Ponatinib inhibits the T670I gatekeeper secondary mutation that sometimes occurs in resistant GIST (Sunitinib and sorafenib also inhibit this mutation). It also inhibits many secondary KIT exon 17 mutations. See links below.
In the 2013 ASCO abstract, the authors concluded: PO potently inhibits the majority of clinically relevant KIT mutant kinases and has a broader spectrum of activity compared to IM (imatinib), SU (sunitinib), or RE (regorafenib). Based on these data, a phase 2 study of PO in drug-resistant GIST is ongoing.



Ponatinib Inhibits Polyclonal Drug-Resistant KIT Oncoproteins and Shows Therapeutic Potential in Heavily Pretreated Gastrointestinal Stromal Tumor (GIST) Patients (full text)


Ponatinib - Ariad homepage


ASCO -2013 - Use of ponatinib to inhibit kinase mutations associated with drug-resistant GIST (abstract) - First author, Michael Heinrich


AACR 2013 - Ponatinib,potently inhibits key activating and drug-resistant KIT mutants found in GIST


FDA Drug Safety Communication: FDA asks manufacturer of the leukemia drug Iclusig (ponatinib) to suspend marketing and sales


ARIAD Announces Temporary Suspension of Iclusig Marketing in the United States Read more: ARIAD Announces Temporary Suspension of Iclusig Marketing in the United States - FierceBiotech http://www.fiercebiotech.com/press-releases/ariad-announces-temporary-suspension-iclusig-marketing-united-states#ixzz2jKdT3Fwo ARIAD Announces Temporary Suspension of Iclusig Marketing in the United States


OncologySTAT - Aug, 6th, 2012 - Ariad prepares for early launch of CML drug ponatinib (may require registration to view article)


Ponatinib prescribing information (PDF)


Ponatinib is active against imatinib-resistant mutants of FIP1L1-PDGFRA and KIT, and against FGFR1-derived fusion kinases


Novel agent ponatinib shows promise in resistant CML


ARIAD PASS program - Patient support program, helps patients to access, afford and adhere (to) medications

Trials of this drug


A Phase 2 Trial of Ponatinib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor


POETIG Trial - POnatinib After rEsisTance to Imatinib in GIST (POETIG)

Trial results

  2014 ASCO - A phase 2 study of ponatinib in patients (pts) with advanced gastrointestinal stromal tumors (GIST) after failure of tyrosine kinase inhibitor (TKI) therapy: Initial report.

Search for this drug/combination in: