Specialty Drugs: Year in Review 2017 Part II

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Specialty Pharmacy Times, January/February, Volume 9, Issue 1

The second part of a comprehensive review of the specialty pharmaceuticals approved by the FDA in 2017.

As we head into 2018, we’ve assembled the second part of our annual review of specialty pharmacy—related FDA approvals and expanded indications that took place over the past year. The first installment reviewed non-oncology specialty actions in 2017, while this installment reviews approvals related to oncology drugs and late-breaking FDA actions that took place after the first installment was published in November last year. Please consult the respective product prescribing monographs for complete information, including dosing regimens.

New oncology molecular entities that were approved in 2017 are detailed below and previously approved oncology drugs that received expanded indications in 2017 are listed in the Table.

Leukemia and Lymphoma

On April 25, 2017, Xatmep (methotrexate) was approved by the FDA for the treatment of pediatric patients with acute lymphoblastic leukemia as a component of a combination chemotherapy maintenance regimen and for the management of pediatric patients with active polyarticular juvenile idiopathic arthritis (pJIA) who are intolerant of or had an inadequate response to first-line therapy. Xatmep is a folate analog metabolic inhibitor available in a ready-to-use oral solution of methotrexate that eliminates the need for needles and the crushing of tablets and compounding into a liquid formulation. Prior to dispensing, Xatmep should be refrigerated (2°C-8°C/36°F-46°F), and after dispensing, it may be stored at room temperature (20°C-25°C/68°F-77°F) for up to 60 days, with excursions permitted to 15°C to 30°C (59°F-86°F). The recommended starting dose in multi-agent combination chemotherapy maintenance regimens is 20 mg/m2 once weekly and, for pJIA, 10 mg/m2 once weekly. Patients should be advised that the recommended dose is once per week and that mistaken daily dosing of Xatmep has led to fatal toxicity.63,64

On April 28, 2017, Rydapt (midostaurin) was approved by the FDA for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults with an fms-like tyrosine kinase 3 (FLT3 gene) mutation and for the treatment of advanced systemic mastocytosis, which includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). Rydapt is approved for use with a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay, which is used to detect the FLT3 mutation in patients with AML. Rydapt is a kinase inhibitor and is available in a 25-mg capsule. The recommended dose for Rydapt in patients with AML is 50 mg orally twice daily with food and, for those with ASM, SM-AHN, or MCL, 100 mg orally twice daily with food.65,66

On June 22, 2017, Rituxan Hycela (rituximab and hyaluronidase human) was approved by the FDA for the treatment of adult patients with previously untreated and relapsed or refractory follicular lymphoma (FL), previously untreated diffuse large B-cell lymphoma (DLBCL), and previously untreated and previously treated chronic lymphocytic leukemia (CLL). Rituxan Hycela is a combination of rituximab (a CD20-directed cytolytic antibody) and hyaluronidase human (an endoglycosidase). This new treatment includes the same monoclonal antibody as intravenous (IV) Rituxan (rituximab) in combination with hyaluronidase human, an enzyme that helps to deliver rituximab under the skin. Treatment should begin only after patients have received at least 1 full dose of a rituximab product by IV infusion, and Rituxan Hycela is not indicated for the treatment of nonmalignant conditions. The recommended dose for FL/DLBCL is 1400 mg/23,400 units (1400 mg rituximab and 23,400 units hyaluronidase) and for CLL is 1600 mg/26,800 units (1600 mg rituximab and 26,800 units hyaluronidase) subcutaneously according to the dosing schedule outlined in the prescribing information. Rituxan Hycela is available as a solution of 1400 mg of rituximab and 23,400 units of hyaluronidase human per 11.7 mL (120 mg/2000 units per mL) and a solution of 1600 mg of rituximab and 26,800 units of hyaluronidase human per 13.4 mL (120 mg/2000 units per mL) in a single-dose vial.67,68

On August 1, 2017, Idhifa (enasidenib) was approved by the FDA for the treatment of patients with relapsed or refractory AML with an isocitrate dehydrogenase 2 (IDH2 gene) mutation as detected by an FDA-approved test. Idhifa is approved for use with a companion diagnostic, the Real Time IDH2 Assay, which is used to detect specific mutations in the IDH2 gene in patients with AML. Idhifa was granted Priority Review and Orphan Drug designations. It is an IDH2 inhibitor that is available in 50-mg and 100-mg tablets. The recommended dose of Idhifa is 100 mg orally once daily until disease progression or unacceptable toxicity.69,70

On August 3, 2017, Vyxeos (cytarabine and daunorubicin) received FDA approval for the treatment of adults with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes. Vyxeos was granted Priority Review, Breakthrough Therapy, and Orphan Drug designations by the FDA. It is available as a fixed-combination medication consisting of 2 chemotherapy drugs: 44 mg daunorubicin (an anthracycline topoisomerase inhibitor) and 100 mg cytarabine (a nucleoside metabolic inhibitor encapsulated in liposomes as a lyophilized cake in a single-dose vial for reconstitution). The recommended induction dose of Vyxeos (daunorubicin 44 mg/m2 and cytarabine 100 mg/m2) liposome via IV infusion is more than 90 minutes on days 1, 3, and 5 and on days 1 and 3 for subsequent cycles of induction if needed. The consolidation dose of Vyxeos (daunorubicin 29 mg/m2 and cytarabine 65 mg/m2) liposome via IV infusion is more than 90 minutes on days 1 and 3.71,72

On August 17, 2017, Besponsa (inotuzumab ozogamicin) was approved by the FDA for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Besponsa was granted Priority Review, Breakthrough Therapy, and Orphan Drug designations by the FDA. It is a CD22-directed antibody—drug conjugate available as a 0.9-mg lyophilized powder in a single-dose vial for reconstitution and further dilution by IV administration. The recommended dosing regimen for cycle 1 and subsequent cycles is dependent on the patient’s response to treatment as outlined in the dosage and administration section of the prescribing information.73,74

On August 30, 2017, Kymriah (tisagenlecleucel) was approved by the FDA for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse. Kymriah is a CD19-directed genetically modified autologous T-cell immunotherapy available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS), called the Kymriah REMS, because of the risks of cytokine release syndrome and neurological events associated with the medication. The FDA granted the drug Priority Review and Breakthrough Therapy designations. It is available in single-dosed units containing 0.2 to 5.0 × 106 chimeric antigen receptor (CAR)—positive viable T cells per kg of body weight for patients 50 kg or less. For patients weighing more than 50 kg, single-dose units containing 0.1 to 2.5 × 108 CAR-positive viable T cells are available. Kymriah is to be administered intravenously via patient-specific infusion bags.75,76

On September 1, 2017, Mylotarg (gemtuzumab ozogamicin) was approved by the FDA for the treatment of newly diagnosed CD33-positive AML in adults and the treatment of relapsed or refractory CD33-positive AML in adults and in pediatric patients 2 years and older. Mylotarg is a CD33-directed antibody—drug conjugate that was granted Orphan Drug designation for its indications. It originally received accelerated approval in May 2000 as a stand-alone treatment for older patients with CD33-positive AML who had experienced a relapse. Mylotarg was voluntarily withdrawn from the market after subsequent confirmatory trials failed to verify clinical benefit and demonstrated safety concerns, including a high number of early deaths. The September 2017 approval includes a lower recommended dose, a different schedule in combination with chemotherapy or on its own, and a new patient population. Mylotarg is available as a 4.5-mg lyophilized cake or powder in a single-dose vial for IV administration. The recommended dose of Mylotarg is dependent on the disease state and treatment experience, which is available in the prescribing information.77-79

On September 14, 2017, Aliqopa (copanlisib) was approved for the treatment of adults with relapsed FL who have received at least 2 prior treatments known as systemic therapies. Continued approval for this indication of Aliqopa—which was granted accelerated approval—may be contingent on verification and description of clinical benefit in a confirmatory trial. The drug also received Priority Review and Orphan Drug designations by the FDA. Aliqopa is a kinase inhibitor available in a 60-mg single-dose lyophilized solid vial for reconstitution. The recommended dose is 60 mg administered as a 1-hour IV infusion on days 1, 8, and 15 of a 28-day treatment cycle on an intermittent schedule (3 weeks on and 1 week off).80,81

On October 18, 2017, Yescarta (axicabtagene ciloleucel) was approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after 2 or more lines of systemic therapy, including DLBCL not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from FL. The FDA granted the drug Priority Review, Breakthrough Therapy, and Orphan Drug designations. It is a CD19-directed genetically modified autologous T-cell immunotherapy available as a cell suspension for infusion. CAR-positive viable T-cell therapy is manufactured specifically for each individual patient. Yescarta’s availability and target dose is 2 × 106 CAR-positive viable T cells per kg of body weight, with a maximum of 2 × 108 CAR-positive viable T cells in approximately 68 mL. Because of the risk of cytokine release syndrome and neurologic toxicities, Yescarta was approved with a REMS. It is to be administered in a certified health care facility.82,83

On October 31, 2017, Calquence (acalabrutinib) was approved by the FDA for the treatment of adult patients with mantle cell lymphoma who have received at least 1 prior therapy. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent on verification and description of clinical benefit in confirmatory trials. The FDA granted the drug Priority Review, Breakthrough Therapy, and Orphan Drug designations. Calquence is a kinase inhibitor available in a 100-mg capsule. The recommended dose is 100 mg orally approximately every 12 hours until disease progression or unacceptable toxicity. Calquence capsules can be swallowed whole with water and with or without food.84,85

Oncology — Breast Cancer

On July 17, 2017, Nerlynx (neratinib) was approved by the FDA for the extended adjuvant treatment of adult patients with early-stage human epidermal growth factor 2 (HER2)—overexpressed/amplified breast cancer to follow adjuvant trastuzumab-based therapy. Nerlynx is a kinase inhibitor available in a 40-mg tablet. The recommended dose is 240 mg (6 tablets) given orally once daily with food, continuously for 1 year. Antidiarrheal prophylaxis is recommended during the first 2 cycles (56 days) of treatment and should be initiated with the first dose.86,87

On March 13, 2017, Kisqali (ribociclib) was approved by the FDA for the combination treatment of postmenopausal women with hormone receptor (HR)—positive, HER2-negative advanced or metastatic breast cancer. The FDA granted this application Priority Review and Breakthrough Therapy designations. Kisqali is a kinase inhibitor available as a 200-mg tablet. The recommended starting dose is 600 mg orally (three 200-mg tablets) taken once daily with or without food for 21 consecutive days followed by 7 days off treatment, resulting in a complete cycle of 28 days. Kisqali should be coadministered with letrozole 2.5 mg once daily throughout the 28-day cycle.88,89

On September 28, 2017, Verzenio (abemaciclib) was approved in combination with fulvestrant for the treatment of women with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy. Verzenio was also approved as monotherapy for the treatment of adult patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting. Verzenio is a selective ATP-competitive inhibitor of cyclin-dependent kinases 4 and 6. It is available for oral use in 50-, 100-, 150-, and 200-mg tablets. The recommended starting dose in combination with fulvestrant is 150 mg twice daily, and the starting dose as monotherapy is 200 mg twice daily with or without food.90,91

On December 1, 2017, Ogivri (trastuzumab-dkst) was approved as a biosimilar to Herceptin for the treatment of patients with breast or metastatic stomach cancer (gastric or gastroesophageal junction adenocarcinoma) whose tumors overexpress the HER2 gene (HER2-positive). Ogivri is a HER2/neu receptor antagonist. Patients are selected for therapy based on an FDA-approved companion diagnostic for a trastuzumab product. It is not to be substituted for or with ado-trastuzumab emtansine. Ogivri is available as a 420-mg lyophilized powder in a multidose vial for reconstitution. It is to be administered as an IV infusion over 90 minutes. Please refer to the prescribing monograph for detailed information regarding doses and treatment schedules.92,93

Other Oncology and Oncology Support

On March 23, 2017, Bavencio (avelumab) was approved by the FDA for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma. The FDA granted the drug Priority Review, Breakthrough Therapy, and Orphan Drug designations. Furthermore, on May 9, 2017, Bavencio received an expanded indication by the FDA for the treatment of patients with locally advanced or metastatic urothelial carcinoma whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. The above indications were granted accelerated approval based on tumor response and duration of response. Continued approval of the above indications may be contingent on verification and description of clinical benefit in confirmatory trials. Bavencio is an antibody that blocks programmed death-ligand 1 (PD-L1) and is available as a 200-mg/10-mL (20 mg/mL) solution in a single-dose vial for IV use. The recommended dose is 10 mg/kg as an IV infusion over 60 minutes every 2 weeks.94-96

On March 27, 2017, Zejula (niraparib) was approved by the FDA for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Zejula is a poly ADP-ribose polymerase (PARP) inhibitor available as 100-mg capsules. It was the first PARP inhibitor to be approved by the FDA that does not require BRCA mutation or other biomarker testing. The FDA granted this application Fast Track, Priority Review, and Breakthrough Therapy designations, as well as Orphan Drug designation specifically for treating recurrent epithelial ovarian cancer. The recommended dose is 300 mg taken once daily with or without food.97,98

On April 28, 2017, Alunbrig (brigatinib) was approved by the FDA for the treatment of patients with anaplastic lymphoma kinase—positive metastatic NSCLC who progressed on or are intolerant to crizotinib. This indication was granted accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent on verification and description of clinical benefit in a confirmatory trial. Alunbrig is a kinase inhibitor available as 30- and 90-mg tablets. The recommended dose is 90 mg orally once daily, with or without food, for the first 7 days. If tolerated, the dose may be increased to 180 mg orally once daily.99,100

On May 1, 2017, Imfinzi (durvalumab) was approved for the treatment of patients with locally advanced or metastatic urothelial carcinoma who had disease progression during or following platinum-containing chemotherapy or who had disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication was granted accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent on verification and description of clinical benefit in confirmatory trials. Imfinzi is a PD-L1—blocking antibody available as a 500-mg/10-mL (50 mg/mL) or 120-mg/2.4-mL (50-mg/mL) solution in a single-dose vial for IV use. The recommended dose is 10 mg/kg as an IV infusion over 60 minutes every 2 weeks.101,102

On September 14, 2017, Mvasi (bevacizumab-awwb), a biosimilar to Avastin (bevacizumab), received FDA approval for the treatment of multiple types of cancer: metastatic colorectal cancer, with IV 5-fluorouracil—based chemotherapy for first- or second-line treatment; metastatic colorectal cancer, with fluoropyrimidine-irinotecan- or fluoropyrimidine-oxaliplatin-based chemotherapy for second-line treatment in patients who have progressed on a first-line bevacizumab product–containing regimen; nonsquamous NSCLC, with carboplatin and paclitaxel for first-line treatment of unresectable, locally advanced, recurrent, or metastatic disease; glioblastoma, as a single agent for adult patients with progressive disease following prior therapy; metastatic renal cell carcinoma with interferon alpha; and cervical cancer, in combination with paclitaxel and cisplatin or paclitaxel and topotecan in persistent, recurrent, or metastatic disease. Mvasi is an anti–vascular endothelial growth factor A monoclonal antibody. It is to be administered only as an IV infusion and not to be initiated for 28 days following a major surgery and until surgical wounds are fully healed. The first infusion is to be administered over 90 minutes, and subsequent infusion times can range from 30 to 60 minutes depending on patient tolerability to first infusion. Recommended dosing and scheduling vary depending on indication. Mvasi is available as a 100-mg/4-mL or 400-mg/16-mL single-dose vial of preservative-free solution.103,104

On November 9, 2017, the FDA approved Cinvanti (aprepitant) in combination with other antiemetic agents for the prevention of acute and delayed nausea and vomiting in adults associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC), including high-dose cisplatin, and nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC). Cinvanti is a substance P/neurokinin-1 receptor antagonist available as a 130-mg injectable emulsion in a single-dose vial. The recommended dose in adults for HEC (single-dose regimen) is 130 mg on day 1 as an IV infusion over 30 minutes approximately 30 minutes prior to chemotherapy. The recommended dosage in adults for MEC (3-day regimen) is 100 mg administered on day 1 as an IV infusion over 30 minutes approximately 30 minutes prior to chemotherapy, and aprepitant capsules (80 mg) are given orally on days 2 and 3. Cinvanti is part of a regimen that includes a corticosteroid and a serotonin type 3 antagonist.105,106

Additional Late-Breaking Specialty FDA Actions

On October 13, 2017, Stelara (ustekinumab) received an expanded indication for the treatment of adolescents (12 years and older) with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. Stelara is a human interleukin-12 and -23 antagonist that was originally approved in September 2009. It is also indicated for the treatment of adult patients with moderate to severe psoriasis who are candidates for phototherapy or systemic therapy; active psoriatic arthritis (PsA), alone or in combination with methotrexate; and moderately to severely active Crohn disease who have failed or were intolerant to treatment with immune modulators or corticosteroids but who never failed a tumor necrosis factor (TNF) blocker or who failed or were intolerant to treatment with 1 or more TNF blockers. Stelara is available as a subcutaneous injection in a 45-mg/0.5-mL or 90-mg/mL single-dose prefilled syringe and a 45-mg/0.5-mL single-dose vial. It is also available as an IV infusion in a 130-mg/26-mL (5-mg/ml) solution in a single-dose vial. The recommended dose for adolescent patients with moderate to severe psoriasis is weight based, and doses are administered subcutaneously at weeks 0 and 4 and then every 12 weeks thereafter.107,108

On October 20, 2017, Simponi Aria (golimumab) received an expanded indication by the FDA for the treatment of adult patients with active PsA or active ankylosing spondylitis (AS). Simponi Aria is a TNF blocker that was originally approved in July 2013. It is also approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis in combination with methotrexate. The recommended dose is a 2 mg/kg IV infusion over 30 minutes at weeks 0 and 4 and then every 8 weeks. For patients with PsA or AS, Simponi Aria may be given with or without methotrexate or other nonbiologic disease-modifying antirheumatic drugs. Corticosteroids, nonsteroidal anti-inflammatory drugs, and/or analgesics may be continued during treatment. Simponi Aria is available as a 50-mg/4-mL (12.5-mg/mL) solution in a single-use vial.109,110

On October 23, 2017, Soliris (eculizumab) received an expanded indication for the treatment of patients with generalized myasthenia gravis (gMG) who are anti—acetylcholine receptor antibody–positive. Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Therefore, it is available only through a restricted program under a REMS. Soliris is a complement inhibitor that was first approved in March 2007. It is also indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria to reduce hemolysis and the treatment of patients with atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy. Soliris is not indicated for the treatment of patients with hemolytic uremic syndrome related to Shiga toxin–producing Escherichia coli. The recommended dose for patients with gMG is 900 mg weekly for the first 4 weeks, followed by 1200 mg for the fifth dose 1 week later, and then 1200 mg every 2 weeks thereafter. Soliris should be administered at the recommended dosage regimen time points or within 2 days of these time points. Soliris is available as a 300-mg/30-mL (10-mg/mL) solution in a single-dose vial for injection.111,112

On November 14, 2017, Fasenra (benralizumab) was approved by the FDA as an add-on maintenance treatment for patients with severe asthma 12 years and older and with an eosinophilic phenotype. Fasenra is an interleukin-5 receptor, alpha-directed cytolytic monoclonal antibody. It is available as a 30-mg/mL solution in a single-dose prefilled syringe for subcutaneous injection. The recommended dose is 30 mg every 4 weeks for the first 3 doses, followed by once every 8 weeks thereafter.113,114

On November 15, 2017, the FDA approved Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (Sly syndrome) in pediatric and adult patients. The FDA granted Mepsevii Fast Track and Orphan Drug designations and a rare pediatric disease priority review voucher for this approval. It is a recombinant human lysosomal beta glucuronidase available in a 10-mg/5-mL (2-mg/mL) single-dose vial for intravenous use. The recommended dose is 4 mg/kg administered every 2 weeks as an intravenous infusion over approximately 4 hours.115,116

On November 16, 2017, Hemlibra (emicizumab-kxwh) was approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. The FDA granted Hemlibra Priority Review, Breakthrough Therapy, and Orphan Drug designations for this approval. It is a bispecific factor IXa— and factor X–directed antibody available in 30-mg/mL, 60-mg/0.4 mL, 105-mg/0.7 mL, and 150-mg/mL single-dose vials for subcutaneous use. The recommended dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly.117,118

On November 21, 2017, Juluca (dolutegravir and rilpivirine) was approved as a complete regimen for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable antiretroviral regimen for at least 6 months with no history of treatment failure and no known substitutions associated with resistance to the individual components of Juluca. Juluca is a 2-drug combination of dolutegravir, an HIV-1 integrase strand transfer inhibitor, and rilpivirine, an HIV-1 non-nucleoside reverse transcriptase inhibitor. It is available as a tablet formulation consisting of 50 mg of dolutegravir (equivalent to 52.6 mg dolutegravir sodium) and 25 mg of rilpivirine (equivalent to 27.5 mg rilpivirine hydrochloride). The recommended dose is 1 tablet taken orally once daily with a meal.119,120

On November 29, 2017, Isentress (raltegravir) received an expanded approval by the FDA for use in combination with other antiretroviral agents for the treatment of HIV-1 infection in pediatric patients weighing at least 2 kg. Isentress is an HIV integrase strand transfer inhibitor. It was originally approved in October 2007 for the treatment of adults with HIV-1 infection in combination with other antiretroviral agents. Isentress is available as 400- and 600-mg film-coated tablets, 100-mg scored and 25-mg chewable tablets, and 100-mg single-use packets for oral suspension. Please refer to the prescribing monograph for weight-based dosing for pediatric patients.121,122

On December 1, 2017, Repatha (evolocumab) received an expanded indication by the FDA to reduce the risk of myocardial infarction, stroke, and coronary revascularization in adults with established cardiovascular disease and is to be used as an adjunct to diet, alone, or in combination with other lipid-lowering therapies (eg, statins, ezetimibe), for treatment of adults with primary hyperlipidemia (including heterozygous familial hypercholesterolemia) to reduce low-density lipoprotein cholesterol (LDL-C).Repatha is a proprotein convertase subtilisin/kexin type 9 inhibitor antibody originally approved in August 2015. It is also indicated as an adjunct to diet and other LDL-lowering therapies (eg, statins, ezetimibe, LDL apheresis) in patients with homozygous familial hypercholesterolemia who require additional lowering of LDL-C. Repatha is available as a single-use 140-mg/mL prefilled SureClick autoinjector or a prefilled syringe and as a 420-mg/3.5-mL solution in a single-use Pushtronex system for subcutaneous use that patients can self-administer. The recommended dosage for adults with established cardiovascular disease or primary hyperlipidemia (including heterozygous familial hypercholesterolemia) is 140 mg every 2 weeks or 420 mg once monthly subcutaneously in the abdomen, thigh, or upper arm.123,124

On December 1, 2017, Taltz (ixekizumab) received an expanded indication by the FDA for the treatment of adults with PsA. Taltz is a humanized interleukin-17A antagonist that was first approved in March 2016 for the treatment of adults with moderate to severe plaque Ps who are candidates for systemic therapy or phototherapy. Taltz is available as a subcutaneous injection in a prefilled auto-injector or as a single-dose prefilled syringe containing 80 mg/mL. The recommended starting dose for PsA is 160 mg (two 80 mg subcutaneous injections) at week 0, followed by 80 mg every 4 weeks. Taltz may be administered alone or in combination with a conventional disease-modifying antirheumatic drug (eg, methotrexate).125,126

On December 12, 2017, Nucala (mepolizumab) received an expanded approval by the FDA for treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). The FDA granted it Priority Review and Orphan Drug designations for this indication. Nucala was first approved in November 2015 as an add-on maintenance treatment for patients with severe asthma 12 years and older and with an eosinophilic phenotype. It is a humanized interleukin-5 antagonist monoclonal antibody (IgG1 kappa) that is available in a single-dose vial containing 100 mg of lyophilized powder for reconstitution. Nucala is not to be used for treatment of other eosinophilic conditions and not for relief of acute bronchospasm or status asthmaticus. The recommended dose for EGPA is 300 mg as 3 separate 100-mg injections administered subcutaneously once every 4 weeks by a health care professional into the upper arm, thigh, or abdomen as detailed in the prescribing information.127,128

On December 13, 2017, Ixifi (infliximab-qbtx) was approved by the FDA as a biosimilar to Remicade. The FDA has approved Ixifi as a treatment for patients with rheumatoid arthritis, Crohn disease, pediatric Crohn disease, ulcerative colitis, AS, PsA, and plaque psoriasis. It is a TNF blocker available as 100 mg of lyophilized infliximab-qbtx in a 15-mL vial for intravenous infusion over a period of not less than 2 hours. Refer to the prescribing monograph for information regarding disease-specific dosage details.129,130

On December 19, 2017, Luxturna (voretigene neparvovec-rzyl) received FDA approval for the treatment of patients with confirmed biallelic RPE65 mutation—associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s). Luxturna received Priority Review, Breakthrough Therapy, and Orphan Drug designations for this approval. It is an adeno-associated virus vector-based gene therapy available as a suspension for subretinal injection, supplied in a 0.5-mL extractable volume in a single-dose 2-mL vial for a single administration in 1 eye. The supplied concentration (5 × 1012 vg/mL) requires a 1:10 dilution prior to administration. The recommended dose for each eye is 1.5 × 1011 vector genomes, administered by subretinal injection in a total volume of 0.3 mL. Subretinal administration should be performed to each eye on separate days within a close interval, with at least 6 days between surgical procedures. It is also recommended to give systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day (maximum of 40 mg/day) for a total of 7 days (starting 3 days before administration of Luxturna to each eye), followed by a tapering dose during the following 10 days.131,132

On December 21, 2017, Siklos (hydroxyurea) was approved by the FDA to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients 2 years and older with sickle cell anemia with recurrent moderate to severe painful crises. The FDA granted Priority Review and Orphan Drug designations for this indication. Siklos is an antimetabolite available as 100-mg and triple-scored 1000-mg tablets. The initial recommended dose is 20 mg/kg once daily. It is important to monitor blood counts on a biweekly basis. The dose may be increased by 5 mg/kg/day every 8 weeks or sooner if a severe painful crisis occurs, until a maximum tolerated dose or 35 mg/kg/day is reached if blood counts are in an acceptable range.133,134

DISCLOSURE

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