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New Drug Update NEW DRUG UPDATE Mark S. Johnson, Pharm.D., BCPS Professor of Pharmacy Practice Director of Postgraduate Education Bernard J. Dunn School of Pharmacy Shenandoah University Financial Disclosures • Mark S. Johnson, Pharm.D., BCPS • Name of commercial interest(s) • Stockholder in Merck, Express Scripts and various mutual funds Objectives • Identify the new drugs approved by the FDA that are most clinically relevant to general practice in the first half of 2018 • Discuss relevant indications, efficacy, pharmacokinetics, safety, and dosing of the new drugs • Discuss how the new drugs differ from existing drugs on the market • Describe how to best incorporate the new drugs into clinical practice using a case-based format Self Assessment Questions 1. Coagulation factor Xa (recombinant), inactivated-zhzo (andexanet alfa) (Andexxa™) is FDA-approved as a reversal agent for which of the following? A. Rivaroxaban (Xarelto™) B. Apixaban (Eliquis™) C. Dabigatran (Pradaxa™) D. Enoxaparin (Lovenox™) 2. Plazomicin (Zemdri™) belongs to which of the following antibiotic classes? A. Quinolones B. Tetracyclines C. Macrolides D. Aminoglycosides Self Assessment Questions 3. Baricitinib (Olumiant™) is a Janus kinase inhibitor that’s FDA-approved for which of the following conditions? A. Psoriasis B. Inflammatory bowel disease C. Rheumatoid arthritis D. Lupus 4. Erenumab-aooe (Aimovig™) is a new drug for migraine prophylaxis. How should it be administered? A. Orally B. Subcutaneously C. Intramuscularly D. Sublingually Self Assessment Questions 5. Sodium Zirconium Cyclosilicate (Lokelma™) is a new drug for treating hyperkalemia. What is its most common adverse effect? A. Hypertension B. Dehydration C. Edema D. Hypercalcemia 6. Tildrakizumab-asmn (Ilumya™) is a new drug for treating psoriasis. What is its mechanism of action? A. Interleukin 12 antagonist B. Interleukin 17 antagonist C. Interleukin 23 antagonist D. TNF inhibitor FDA Approvals • 2018 to date (as of 8/17/18) • 31 New Molecular Entities / New Biolologics • 19 New Dosage Forms • 1 Withdrawal Withdrawals • Daclizumab (Zinbryta™)—Biogen/AbbVie • Humanized monoclonal antibody for relapsing remitting multiple sclerosis • MOA: modulator of interleukin-2-mediated activation of lymphocytes by binding to the alpha subunit CD25 of the interleukin-2 receptor • Manufacturer voluntarily removed from the worldwide market on March 2, 2018 due to safety concerns • Severe liver damage including autoimmune hepatitis and liver failure • Immune-related disorders including skin reactions, lymphadenopathy, non-infectious colitis, other immune- mediated disorders SIGNIFICANT NEW MOLECULAR ENTITIES/BIOLOGICS Cardiovascular Drugs Coagulation factor Xa (recombinant), inactivated-zhzo (andexanet alfa) (Andexxa™): Portola Pharm • Anticoagulation reversal agent for the direct factor Xa inhibitors rivaroxaban (Xarelto™) and apixaban (Eliquis™) when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding • First reversal agent for the factor Xa inhibitors (but not for edoxaban [Savaysa™] or betrixaban [Bevyxxa™] • Idarucizumab (Praxbind™) was approved in 2015 for reversal of the anticoagulant effect of the direct thrombin inhibitor dabigatran (Pradaxa™) • Studies suggest that the anticoagulant effect of factor Xa inhibitors may be reversed by prothrombin complex concentrates as well https://www.empr.com Coagulation Factor Xa (Andexxa™) MOA • Genetically modified variant of human factor Xa (alanine substituted for serine) produced in Chinese hamster ovary cells • Exerts procoagulant effect by binding and sequestering the Factor Xa inhibitors rivaroxaban and apixaban • Also exerts procoagulant effect by binding and inhibiting the activity of Tissue Factor Pathway Inhibitor, which can lead to increase tissue factor-initiated thrombin generation https://www.nejm.org/doi/full/10.1056/NEJMoa1510991 Coagulation Factor Xa (Andexxa™) Efficacy • FDA approval based on 2 randomized, placebo-controlled trials in healthy volunteers 50-75yo (ANNEXA-A and ANNEXA-R) • Evaluated mean change from baseline in anti-factor Xa activity following administration of andexanet alfa • Patients had received either apixaban or rivaroxaban • In phase 2 trials and animal studies, andexanet alfa shown to reduce the anti-factor Xa activity of other direct or indirect factor Xa inhibitors • FDA requiring Portola Pharm to conduct a randomized, controlled trial comparing andexanet alfa with usual care (including prothrombin complex concentrates) in patients taking factor Xa inhibitors with active bleeding Coagulation Factor Xa (Andexxa™) Efficacy: ANNEXA-A • N=66 healthy subjects • Received apixaban 5mg twice daily for 3.5 days • 3 hours after the last dose of apixaban andexanet alfa 400-mg IV bolus with or without a subsequent 4 mg/min continuous infusion for 2 hours vs. placebo • Anti-factor Xa activity reduced within 2-5 minutes by 94% with an andexanet alfa IV bolus vs. 21% placebo • Thrombin generation was fully restored within 2-5 minutes in 100% of andexanet-treated patients vs. 11% placebo • Effects sustained throughout the continuous 2h infusion Coagulation Factor Xa (Andexxa™) Efficacy: ANNEXA-R • N=80 healthy subjects • Received rivaroxaban 20mg once daily for 4 days • 4 hours after the last dose of rivaroxaban andexanet alfa 800-mg IV bolus with or without a subsequent 8 mg/min continuous infusion for 2 hours vs. placebo • Anti-factor Xa activity reduced within 2-5 minutes by 92% with an andexanet alfa IV bolus vs. 18% placebo • Thrombin generation was fully restored within 2-5 minutes in 96% of andexanet-treated patients vs. 7% placebo • Effects sustained throughout the continuous 2h infusion Coagulation Factor Xa (Andexxa™) Efficacy: ANNEXA-4 Interim Analysis • Ongoing single-arm trial • N=238 patients with acute major bleeding (61% intracerebral, 27% gastrointestinal) • Received andexanet alfa (89% low-dose regimen and 11% high-dose regimen) within 18h after taking factor Xa inhibitor (apixaban, rivaroxaban, edoxaban, enoxaparin) • Reduction of anti-factor Xa activity by 91% apixaban, by 88% rivaroxaban, by 75% enoxaparin • Effective hemostasis achieved in 83% of patients within 12h Coagulation Factor Xa (Andexxa™) Safety • Warnings/Precautions • Arterial and venous thromboembolic events, ischemic events, and cardiac events, including sudden death, have occurred during treatment • Interim analysis of ANNEXA-4: 11% of patients had thrombotic event and 12% died within 30 days with median time to the first event of 6 days • No thromboembolic events in 223 healthy volunteers • Resume anticoagulant therapy as soon as medically appropriate following treatment • Re-elevation or incomplete reversal of anticoagulant activity can occur • ADR’s • Most common adverse reactions (≥ 5%): urinary tract infections and pneumonia • Most common adverse reactions (≥ 3%) in healthy volunteers: infusion-related reactions Coagulation Factor Xa (Andexxa™) Availability/Dosing/Cost • Availability: Cartons containing four 100-mg single-use vials; limited supply until early 2019 • Dosing: Recommended dosage is based on the factor Xa inhibitor taken, its dose, and the time since the last factor Xa inhibitor dose. • Cost: High dose=$49,500; Low dose=$24,750 Coagulation Factor Xa (Andexxa™) Conclusion • Coagulation Factor Xa is an anticoagulation reversal agent for the direct factor Xa inhibitors rivaroxaban and apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding • Can rapidly reverse the anticoagulant effect of apixaban and rivaroxaban in patients with active major bleeding, based on an interim analysis of an ongoing single-arm trial • It may also be effective in reversing the anticoagulant effect of other direct factor Xa inhibitors and indirect factor Xa inhibitors but data are lacking • Arterial and venous thromboembolic events, ischemic events, and cardiac events, including sudden death, have occurred • How it compares with prothrombin complex concentrates is not established • Costly Case • A 65yo F with atrial fibrillation on rivaroxaban (Xarelto™) falls coming down her steps and hits her head. An intracranial bleed is seen on CT scan. How should this patient best be treated? Infectious Diseases Drugs Vancomycin Oral Solution (Firvanq™) Cutis Pharma • A glycopeptide antibacterial indicated in adults and pediatrics < 18yo for treatment of Clostridium difficile- associated diarrhea and enterocolitis caused by Staphylococcus aureus (including MRSA) • Only FDA-approved vancomycin oral solution available • Replaced vancomycin compounding kit (FIRST™ Vancomycin Kit) • Oral capsule formulation (Vancocin™) also available • IV formulation has been used in past to compound the oral solution as less costly than capsules Vancomycin Oral Solution (Firvanq™) Efficacy • No new clinical trials were required for approval of Firvanq™ by the FDA • Approval was based on two trials conducted earlier with Vancocin™ oral capsules showing efficacy for treatment of Clostridium difficile infections • Oral vancomycin now recommended as first line therapy for CDI in 2017 Guidelines Vancomycin Oral Solution (Firvanq™) ADRs • Poorly absorbed, but significant systemic absorption can occur in patients with renal insufficiency and/or colitis who have taken multiple oral doses. • Also some patients with inflammatory disorders of the intestinal mucosa also may have significant systemic absorption • Most common adverse reactions (≥ 10%): • nausea (17%) • abdominal pain (15%) • hypokalemia (13%) Vancomycin Oral Solution
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