updated 5/15/2020

Ayvakit – Approved January 9th, 2020

On January 9, 2020, The FDA approved avapritinib, brand name Ayvakit (formerly BLU-285), for GISTs with a mutation in exon 18 of the platelet derived growth factor receptor alpha (PDGFRA), including the D842V mutation. It is approved for patients in any line of treatment, for example, 1st line, 2nd line, etc., but it is not approved for adjuvant treatment (preventative).

Prior to the approval of Ayvakit, these mutations have not had an approved treatment to which they responded. In the NAVIGATOR trial, the response rate to Ayvakit was 84% for all exon 18 mutations and 89% for the D842V mutation. Response includes complete responses (7% and 8% respectively) and partial responses (77%% and 82%) respectively. The median duration of response has not yet been reached in these patient groups.

Like other drugs approved for GIST, avapritinib is a tyrosine kinase inhibitor (TKI). It inhibits PDGFRA, KIT and CSFR1. It is also being developed for mastocytosis.

The recommended dosage of Ayvakit is 300 mg, given once per day, at least one hour before and two hours after a meal. It is available in 100, 200 and 300 mg tablets.

You may have questions about this treatment. As always, please consult your GIST specialist to determine if this is a potential treatment option for you. Our Patient Registry staff is happy to answer questions about avapritinib and other approved treatment options for GIST. Please contact Sahibjeet Kaur at skaur@liferaftgroup.org

Blueprint Medicines developed avapritinib. Access their press release here: https://www.blueprintmedicines.com/wp-content/uploads/2020/01/Blueprint-Medicines-Announces-FDA-Approval-of-AYVAKIT-Press-Release-January-2020.pdf

The full FDA approval statement on avapritinib is summarized here: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-avapritinib-gastrointestinal-stromal-tumor-rare-mutation

The full prescribing information describes trial results and discussed side effects. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/212608s000lbl.pdf


  • 100 mg, round, white film-coated, printed with blue ink “BLU” on one side and “100” on the other side.
  • 200 mg, capsule shaped, white film-coated, printed with blue ink “BLU” on one side and “200” on the other side.
  • 300 mg, capsule shaped, white film-coated, printed with blue ink “BLU” on one side and “300” on the other side.

Other resources:

For a history of the development and drug approval process for Ayvakit: https://www.drugs.com/history/ayvakit.html

A guide for patients is available on the Ayvakit website: https://ayvakit.com/wp-content/uploads/AYVAKIT_Patient_and_Caregiver_Guide.pdf

Information on the importance of mutational testing can be found in Blueprint’s educational program, Mutation Matters.

For further information on mutations in GIST: http://staging.liferaftgroup.org/mutations-and-mutation-testing/

For helpful resources and for support, contact YourBlueprint, a patient support program designed to assist you during your treatment with Ayvakit: https://www.yourblueprint.com/

For information about avapritinib on the LRG Clinical Trials website: https://gisttrials.org/iLRG/drug_detail.php?drug=321

References on PDGFRA and D842V: http://staging.liferaftgroup.org/2007/12/references-pdgfra_and_d842v/


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