TAFINLAR® (Dabrafenib): The FDA on May 29, 2013 approved the use of TAFINLAR® capsule for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test. TAFINLAR® is not indicated for the treatment of patients with wild-type BRAF melanoma because of the potential risk of tumor proliferation. The FDA also approved the THxID BRAF assay (bioMerieux, Inc.) for detection of BRAF V600E mutations. TAFINLAR® is a product of GlaxoSmithKline, LLC.
Author: RR
XOFIGO® (Radium Ra 223 dichloride)
XOFIGO® (Radium Ra 223 dichloride): The FDA on May 15, 2013 approved XOFIGO® Injection for the treatment of castration-resistant prostate cancer patients with symptomatic bone metastases and no known visceral metastatic disease. XOFIGO® is an alpha-particle emitting radiotherapeutic drug. It mimics calcium and forms complexes with hydroxyapatite at sites of increased bone turnover, as seen at metastatic lesions in the bone. XOFIGO® is a product of Bayer HealthCare Pharmaceuticals Inc.
TARCEVA® (Erlotinib)
TARCEVA® (Erlotinib): The FDA on May 14, 2013 approved TARCEVA® for the first-line treatment of metastatic non-small cell lung cancer (NSCLC) patients whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations. The FDA concurrently approved the cobas EGFR Mutation Test, a companion diagnostic test for detection of these molecular abnormalities. TARCEVA has had prior FDA approval for the treatment of patients with locally advanced or metastatic NSCLC after failure of at least one prior chemotherapy regimen and for maintenance treatment of patients with locally advanced or metastatic NSCLC, whose disease has not progressed after four cycles of platinum-based first-line chemotherapy. TARCEVA® is a product of Astellas Pharma Inc.
MYELOMA – mSMART is the way to go
Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) developed by the Mayo Clinic, unlike the present staging systems takes cytogenetic features into consideration and stratifies patients into High risk, Intermediate risk and Low risk groups. The clinician, based on the risk, then decides on the most appropriate therapy. This Risk-Adapted Therapy should result in better outcomes without compromising care. The 2013 mSMART Consensus Guidelines were published in the April issue of the Mayo Clinic Proceedings.
TTP, HUS and aHUS Different diseases – Different treatments
SUMMARY: Thrombotic Thrombocytopenic Purpura (TTP), Hemolytic Uremic Syndrome (HUS) and Atypical Hemolytic Uremic Syndrome (aHUS) are Thrombotic Microangiopathies (TMA’s) associated with MicroAngiopathic Hemolytic Anemia and thrombocytopenia. Even though their clinical presentation has some similarities, they are distinct entities with different pathophysiology and hence managed differently. With the identification of von Willebrand Factor (vWF) cleaving protease ADAMTS13 (A disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13) in 1996, we are now able to better understand and appropriately manage these TMA’s. Patients with TTP are deficient in ADAMTS13 and therefore develop platelet microthrombin in small blood vessels due to uninhibited propagation of platelet aggregates bound to ultra high molecular weight VWF multimers. Approximately 10% or less of Shiga-Toxin producing Escherichia Coli (STEC) infections may be associated with HUS. aHUS is caused by a genetic deficiency of one or more complement regulatory proteins which results in uncontrolled activity of the alternate complement pathway. Plasma Exchange in TTP restores the protease activity of ADAMTS13 whereas aHUS is treated with SOLIRIS® (Eculizumab) to inhibit complement mediated TMA. Once a diagnosis of STEC-HUS is confirmed, hospitalization and intensive care with transfusions and kidney dialysis may become necessary. George JN. Blood 2010:116; 4060-4069
Efficacy and safety of regorafenib for advanced gastrointestinal stromal tumours after failure of imatinib and sunitinib (GRID) an international, multicentre, randomised, placebo-controlled, phase 3 trial
SUMMARY: STIVARGA® (Regorafenib) is an oral multikinase inhibitor. In this international trial, 199 patients with advanced GIST who had progressed on both GLEEVEC® (Imatinib) and SUTENT® (Sunitinib) were randomized in a 2:1 ratio to receive either STIVARGA® or placebo. The primary endpoint was progression-free survival (PFS). Patients in the STIVARGA® group had a statistically significant 3.9 month improvement in progression-free survival (PFS) compared to placebo (4.8 months vs 0.9 months; Hazard Ratio = 0.27; P<.0001). Following progression, 85% of the patients assigned to the placebo group crossed over to the STIVARGA® group. Treatment was well tolerated and the most common adverse events included hypertension, hand-foot syndrome and diarrhea. There is now a new treatment option for patients with GIST who progress on GLEEVEC® and SUTENT®. Demetri GD, Reichardt P, Kang Y, et al. The Lancet. 2013;381:295-302
STIVARGA® (Regorafenib)
The FDA on February 25, 2013 approved the use of STIVARGA® for the treatment of patients with advanced GastroIntestinal Stromal Tumors (GIST) that are unresectable or metastatic and are no longer responding to GLEEVEC® (Imatinib) and SUTENT® (Sunitinib). The FDA initially approved STIVARGA® in 2012 for the treatment of patients with metastatic colorectal cancer (mCRC) who have been previously treated with fluoropyrimidine, ELOXATIN® (Oxaliplatin), and CAMPTOSAR® (Irinotecan) based chemotherapy, an anti-VEGF therapy, and, if KRAS wild type, an anti-EGFR therapy. STIVARGA® is a product of Bayer HealthCare Pharmaceuticals, Inc.
STIVARGA® (Regorafenib) for advanced GIST
STIVARGA® is an oral multikinase inhibitor. In a study published in the January 2013 issue of THE LANCET, treatment with STIRVAGA® in patient population resistant or refractory to GLEEVEC® and SUTENT® resulted in significant improvement in progression free survival. This is the third agent approved by the FDA for advanced GIST. As patients with advanced GIST live longer, newer agents with unique mechanism of action will take center stage.
KADCYLA® (Ado-Trastuzumab Emtansine, T-DM1):
The FDA on February 22, 2013 approved KADCYLA® for the treatment of patients with HER2-positive metastatic breast cancer who have received prior treatment with HERCEPTIN® (Trastuzumab) and a Taxane chemotherapy. KADCYLA® is marketed by Genentech U.S., Inc.