FDA Approves Avapritinib for the Treatment of Adult Patients with Indolent Systemic Mastocytosis
Blueprint Medicines is pleased to announce that AYVAKIT® (avapritinib) is now approved by the US Food and Drug Administration (FDA) for the treatment of adult patients with indolent systemic mastocytosis (ISM). ISM is a rare mast cell disease that can lead to the proliferation and activation of abnormal mast cells which can affect multiple organ systems.1,2 ISM is driven by the KIT D816V mutation in about 95% of cases. It can be characterized by a range of symptoms, including skin, gastrointestinal, neurocognitive, and systemic symptoms (including anaphylaxis).2,6-8.
AYVAKIT is a tyrosine kinase inhibitor designed for the potent and selective inhibition of KIT D816V—the only FDA-approved treatment to selectively target the underlying driver of disease in ~95% of patients with ISM.3-5,9.
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM).
Limitations of Use: AYVAKIT is not recommended for patients with platelet counts of <50 x 109/L.
IMPORTANT SAFETY INFORMATION
There are no contraindications for AYVAKIT.
Intracranial Hemorrhage (ICH)—Serious ICH may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. No events of ICH occurred in the 246 patients with ISM who received any dose of AYVAKIT in the PIONEER study. Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status.
Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs.
Cognitive Effects—Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC; <1% were Grade 3. Depending on the severity, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT.
Photosensitivity—AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.
Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose.
Adverse Reactions—The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.
Drug Interactions—Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors or inducers. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here to see the full Prescribing Information for AYVAKIT.
Novartis ribociclib only Category 1 preferred first-line treatment option for HR+/HER2- mBC in combination with an AI in updated NCCN Clinical Practice Guidelines in Oncology
East Hanover, March 28, 2023 — Updates to the NCCN Guidelines® for breast cancer, released in January 2023, recommend ribociclib (Kisqali®) as the only Category 1 preferred CDK4/6 inhibitor (CDK4/6i) for first-line treatment of patients with hormone receptor-positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer (mBC) when combined with an aromatase inhibitor (AI). This recommendation indicates high levels of clinical evidence and uniform consensus among NCCN on ribociclib (Kisqali) as an appropriate treatment.
NCCN Guidelines also continue to recommend ribociclib (Kisqali) plus fulvestrant as a Category 1 preferred regimen for first- and subsequent-line therapies* in HR+/HER2- mBC.
The latest NCCN Guidelines recommend ribociclib (Kisqali) for demonstrating significant overall survival (OS) benefit in combination with various endocrine therapies across three Phase III MONALEESA trials in HR+/HER2- mBC and uniquely in combination with an AI in the first-line setting in MONALEESA-2. These recent updates to the guidelines reinforce key distinctions among the CDK4/6i in mBC, driving the potential to enhance patient access to the latest evidence-based care and to improve outcomes.
In addition to consistently demonstrating statistically significant OS benefit, Kisqali preserved or improved patients’ quality of life in all three Phase III MONALEESA trials.
For more information read the Novartis announcement.
FDA Approves Polatuzumab Vedotin-piiq for Previously Untreated Diffuse Large B-cell Lymphoma
On April 19, the U.S Food and Drug Administration (FDA) approved polatuzumab vedotin-piiq with a rituximab product, cyclophosphamide, doxorubicin, and prednisone for adult patients who have previously untreated diffuse large B-cell lymphoma, not otherwise specified, or high-grade B-cell lymphoma and who have an International Prognostic Index score of 2 or greater.
For more information, read the FDA announcement and Genentech announcement
FDA Approves Enfortumab Vedotin-ejfv + Pembrolizumab for Locally Advanced or Metastatic Urothelial Carcinoma
On April 3, U.S the Food and Drug Administration (FDA) granted accelerated approval to enfortumab vedotin-ejfv with pembrolizumab for patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy.
For more information read the FDA announcement, the Merck announcement and the Seagen announcement.
FDA Grants Accelerated Approval to Retifanlimab-dlwr for MCC
On March 22, 2023, the U.S Food and Drug Administration granted accelerated approval to retifanlimab-dlwr for adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC).
For more information read the FDA announcement and the Incyte announcement.
FDA Approves Dabrafenib + trametinib for Pediatric Patients with LGG
On March 16, 2023, the U.S Food and Drug Administration (FDA) approved dabrafenib with trametinib for pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.
For more information read the FDA announcement and the Novartis announcement.
FDA Approves Trodelvy® in Pre-treated HR+/HER2- Metastatic Breast Cancer
On February 3, 2023, the U.S. Food and Drug Administration (FDA) approved Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with unresectable locally advanced or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.
Click here to review the data.
Please see full Prescribing Information including BOXED WARNING.
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©2023 Gilead Sciences, Inc. All rights reserved. US-TROP-0782 02/23
FDA Approves Pembrolizumab as Adjuvant Treatment for NSCLC
On January 26, 2023, the U.S Food and Drug Administration (FDA) approved pembrolizumab for adjuvant treatment following resection and platinum-based chemotherapy for stage IB (T2a ≥4 cm), II, or IIIA non-small cell lung cancer (NSCLC).
For more information read the FDA announcement and the Merck announcement.
FDA Approves Zanubrutinib for CLL or SLL
On January 19, 2023, the U.S Food and Drug Administration (FDA) approved zanubrutinib for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
For more information, read the FDA announcement and the Beigene announcement.
FDA Grants Accelerated Approval to Tucatinib with Trastuzumab for Colorectal Cancer
On January 19, 2023, the U.S Food and Drug Administration (FDA) granted accelerated approval to tucatinib in combination with trastuzumab for RAS wild-type HER2-positive unresectable or metastatic colorectal cancer that has progressed following fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy.
For more information read the FDA announcement.