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.
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.
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.
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.
GlaxoSmithKline (GSK) is planning to voluntarily withdraw the indication of niraparib for treatment of adult patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have
been treated with three or more prior chemotherapy regimens and whose cancer is associated with homologous
recombination deficiency positive status.
For more information read the GSK letter.
On August 29, 2022, the Centers for Medicare & Medicaid Services (CMS) published a final rule (CMS-5527-F2) that finalized delaying the Radiation Oncology Model start date until a date is determined through future rulemaking.
For more information, visit the Radiation Oncology Model website.
On August 24, 2022, the U.S Food and Drug Administration (FDA) approved ibrutinib for the treatment of pediatric patients one year and older with chronic graft-versus-host disease after failure of one or more lines of systemic therapy.
For more information, read the FDA announcement.
On August 10, 2022, the U.S. Food and Drug Administration (FDA) granted regular approval to capmatinib for adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation leading to mesenchymal-epithelial transition (MET) exon 14 skipping, as detected by an FDA-approved test.
For more information, read the FDA announcement.
On August 5, 2022, the Food and Drug Administration approved new tablet formulation of acalabrutinib (Calquence, AstraZeneca) under accelerated approval based on overall response rate for all current indications, including adult patients with chronic lymphocytic leukemia, small lymphocytic lymphoma, and patients with relapsed or refractory mantle cell lymphoma.
Read full press release.
Posted on 08/05/2022
On May 27, 2022, the U.S. Food and Drug Administration (FDA) approved both nivolumab in combination with fluoropyrimidine- and platinum-containing chemotherapy and nivolumab plus ipilimumab as a first-line treatment for adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma regardless of programmed death-ligand 1 (PD-L1) status.
For more information, read Bristol Myers Squibb's announcement.
On May 28, 2022, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tisagenlecleucel for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma after two or more lines of systemic therapy.
For more information, read Novartis' announcement.
On March 23, 2022, the U.S. Food and Drug Administration (FDA) approved lutetium Lu 177 vipivotide tetraxetan for the treatment of adult patients with prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer who have been treated with androgen receptor pathway inhibition and taxane-based chemotherapy.
Read the FDA announcement.
Read Novartis' announcement.
On December 3, 2021, the U.S. Food and Drug Administration approved Keytruda® (pembrolizumab) for the adjuvant treatment of adult and pediatric (greater than or equal to 12 years of age) patients with stage IIB or IIC melanoma following complete resection.
Read the FDA announcement.
Read Merck's announcement.