Subject: Ayvakit 3491-A SGM P2021a
Policy:
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are
considered a covered benefit provided that all the approval criteria are met and the member
has no exclusions to the prescribed therapy.
A. FDA-Approved Indications
1. Gastrointestinal Stromal Tumor (GIST)
Ayvakit 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.
2. Advanced Systemic Mastocytosis (AdvSM)
Ayvakit 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).
Limitations of Use: Ayvakit is not recommended for the treatment of patients with
AdvSM with platelet counts of less than 50 X 109/L.
B. Compendial Uses
1. Myeloid/lymphoid neoplasms with eosinophilia and FIP1L1-PDGFRA rearrangement
2. Unresectable, recurrent, or metastatic GIST after failure on approved therapies
All other indications are considered experimental/investigational and not medically
necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization
review:
A. For GIST: PDGFRA exon 18 mutation testing (e.g., polymerase chain reaction [PCR]
-based assay, next-generation sequencing [NGS]-based assay) results (where
applicable).
B. For myeloid and/or lymphoid neoplasms with eosinophilia: Testing or analysis
confirming FIP1L1-PDGFRA rearrangement and PDGFRA D842V mutation
III. CRITERIA FOR INITIAL APPROVAL
A. Gastrointestinal Stromal Tumor (GIST)
Authorization of 12 months may be granted for treatment of gastrointestinal stromal
tumor (GIST) when either of the following criteria are met:
1. The disease harbors a platelet-derived growth factor receptor alpha (PDGFRA) exon
18 mutation, including PDGFRA D842V mutations.
2. The disease is unresectable, recurrent, or metastatic and member has failed at
least four FDA-approved therapies (e.g., imatinib, sunitinib, regorafenib, and
ripretinib).
B. Myeloid/Lymphoid Neoplasms with Eosinophilia
Authorization of 12 months may be granted for treatment of myeloid and/or lymphoid
neoplasms with eosinophilia when all of the following criteria are met:
1. The disease harbors a PDGFRA D842V mutation which is resistant to imatinib.
2. The disease is FIP1L1-PDGFRA rearrangement-positive.
C. Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic
Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematological
Neoplasm (SM-AHN) and Mast Cell Leukemia (MCL)
Authorization of 12 months may be granted for treatment of AdvSM, ASM, SM-AHN
and MCL when the member’s platelet count is greater than or equal to 50 X 109 /L.
IV. CONTINUATION OF THERAPY
A. GIST
Authorization of 12 months may be granted for continued treatment of GIST when the
member is receiving clinical benefit and there is no evidence of generalized
(widespread, systemic) disease progression or unacceptable toxicity while on the
current regimen.
B. Myeloid/Lymphoid Neoplasms with Eosinophilia and AdvSM including ASM,
SM-AHN, MCL
Authorization of 12 months may be granted for continued treatment in members
requesting reauthorization when there is no evidence of unacceptable toxicity or
disease progression while on the current regimen.
Place of Service:
Outpatient
The above policy is based on the following references:
V. REFERENCES
- Ayvakit [package insert]. Cambridge, MA: Blueprint Medicines Corporation; June 2021.
- The NCCN Drugs & Biologics Compendium® © 2020 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed November 2, 2020.
- NCCN Clinical Practice Guidelines in Oncology® Gastrointestinal Stromal Tumors (GIST) (Version 1.2021). © 2020 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed November 2, 2020.
Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.
July 21, 2021
