Oncology Newsfeed

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.

INDICATION

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.

Posted 5/24/2023

FDA Approves Avapritinib for Indolent Systemic Mastocytosis

On May 22, the U.S. Food and Drug Administration (FDA) approved avapritinib for the treatment of adults with indolent systemic mastocytosis.

For more information, read the Blueprint Medicines announcement.

Posted 5/24/2023

FDA Approves Epcoritamab-bysp for DLBCL

On May 19, the U.S. Food and Drug Administration (FDA) granted accelerated approval to epcoritamab-bysp  for the treatment of adult patients with for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from indolent lymphoma and high-grade B-cell lymphoma after two or more lines of systemic therapy.

For more information read the FDA announcement and Genmab announcement.

Posted 5/22/2023

FDA Approves FoundationOne Liquid CDx as Companion Diagnostic for Mobocertinib

On May 4, the U.S. Food and Drug Administration (FDA) approved the FoundationOne^®Liquid CDx to be used as a companion diagnostic for mobocertinib, which is currently FDA-approved for adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations, as detected by an FDA-approved test, and whose disease has progressed on or after platinum-based chemotherapy.

For more information, read the Foundation Medicine announcement. 

Posted 5/4/2023

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

Posted 4/21/2023

FDA Approves Omidubicel for Patients with Hematologic Malignancies

On April 17, the U.S Food and Drug Administration (FDA) approved omidubicel-onlv for use in adult and pediatric patients (12 years of age and older) with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.

For more information read the FDA announcement.

Posted 4/19/2023

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. 

Posted 4/3/2023

FDA Approves Expanded Indication for Illuccix® to Include Patient Selection for PSMA-Directed Radioligand Therapy

On March 16, 2023, The U.S Food and Drug Administration approved a supplementary New Drug Application for Illuccix® (kit for the preparation of gallium Ga 68 gozetotide injection) to enable its use for the selection of patients with metastatic prostate cancer, for whom lutetium-177 (177Lu) PSMA-directed therapy is indicated. 

For more information read the Telix Pharmaceuticals announcement.

Posted 3/29/2023

Immunogen Announces C-code for Mirvetuximab Soravtansine-gynx

Elahare announced the new Healthcare Common Procedure Coding System (HCPCS) C-code for Elahere^TM (mirvetuximab soravtansine-gynx), issued by the Centers for Medicare & Medicaid Services (CMS). The code C9146 is effective April 1, 2023 for mirvetuximab soravtansine-gynx injection of 1mg and may be used on claims billed under the hospital Outpatient Prospective Payment System.

For more information visit the Elahare website. 

Posted 3/23/2023

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. 

Posted 3/22/2023