Specialty Drugs: Year in Review 2014

Stacey Ness, PharmD, RPh, CSP, MSCS, AAHIVP;Ruby Mhajan, RPh;

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February 9, 2015

Specialty Pharmacy Times

January/February 2015

Volume6

Issue 1

Specialty Drugs: Year in Review 2014

Author(s):

Stacey Ness, PharmD, RPh, CSP, MSCS, AAHIVP,Ruby Mhajan, RPh

In 2014, the FDA approved notable new therapies for the treatment of cancer, bleeding disorders, inflammatory conditions, cystic fibrosis, pulmonary fibrosis, HIV, hepatitis C, multiple sclerosis, angioedema, and other conditions.

In 2014, specialty pharmaceuticals featured prominently in the FDA’s new drug approval and expanded indications list. Below is a summary of specialty pharmacy—related FDA approvals and expanded indications for 2014. Please consult product prescribing information for complete information.

Oncology—Leukemias and Lymphomas

On February 12, 2014, Imbruvica (ibrutinib; Janssen) received an expanded indication for the treatment of chronic lymphocytic leukemia (CLL) in patients who had received at least 1 previous therapy. Furthermore, on July 28, 2014, Imbruvica was granted an expanded indication to treat patients with CLL who carry a deletion in chromosome 17, which is typically associated with a poor response to standard treatment for CLL. Imbruvica is an inhibitor of Bruton’s tyrosine kinase. It was initially approved on November 13, 2013, for the treatment of mantle cell lymphoma (MCL). The recommended dosage is 560 mg (four 140-mg capsules) taken orally once daily for MCL, while the recommended dose for CLL is 420 mg (three 140-mg capsules) taken orally once daily.^1-3

Sylvant (siltuximab; Janssen Biotech, Inc) was approved, on April 22, 2014, for patients with multicentric Castleman’s disease (MCD), a rare disorder similar to lymphoma. Sylvant is an interleukin-6 antagonist that works by blocking a protein that stimulates abnormal growth of immune cells. It is intended for patients with MCD who do not have HIV or human herpes virus-8. It is the first FDA-approved drug to treat patients with MCD. Sylvant is available as 100 mg or 400 mg of lyophilized powder in a single-use vial. The recommended dose is 11 mg/kg infused intravenously over 1 hour every 3 weeks.^4,5

Purixan (mercaptopurine; Nova Laboratories) was approved on April 28, 2014, as a new dosage form, an oral suspension. Purixan is a nucleoside metabolic inhibitor indicated for the treatment of patients with acute lymphoblastic leukemia (ALL) as a part of a combination regimen. It is supplied as a 20-mg/ml oral suspension. Mercaptopurine was originally approved and commercially available as a 50-mg tablet in 1953.^6,7

On July 3, 2014, Beleodaq (belinostat; Spectrum Pharmaceuticals, Inc) was approved for patients being treated for relapsed or refractory peripheral T-cell lymphoma, a rare and fast-growing type of non-Hodgkin lymphoma. Beleodaq is a histone deacetylase inhibitor that works by stopping enzymes that contribute to T-cells becoming cancerous. Beleodaq is available as 500 mg of lyophilized powder for reconstitution in a single-use vial, and the recommended dose is 1000 mg/m² administered over 30 minutes by IV infusion once daily on days 1 through 5 of a 21-day cycle. Cycles can be repeated until disease progression or unacceptable toxicity.^8,9

Zydelig (idelalisib; Gilead Sciences, Inc) was approved on July 23, 2014, for 3 types of blood cancers. It was approved for relapsed CLL, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other comorbidities; in patients with relapsed follicular B-cell non-Hodgkin lymphoma (FL) who have received at least 2 prior systemic therapies; and in patients with relapsed small lymphocytic lymphoma (SLL) who have received at least 2 prior systemic therapies. Zydelig is a kinase inhibitor that has been approved to treat FL and SLL under the FDA’s accelerated approval program. Zydelig is available in 150-mg and 100-mg tablets, and the recommended starting dose is 150 mg orally twice daily.^10,11

Blincyto (blinatumomab; Amgen Inc) was approved on December 3, 2014, for the treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor ALL. Blincyto is a bispecific CD19-directed CD3 T-cell engager and was approved under the accelerated approval program. Blincyto is the first approved drug that engages the body’s T cells to destroy leukemia cells. Blincyto is available as 35 mcg of lyophilized powder in a single-use vial for reconstitution. The medication is to be administered as a continuous intravenous (IV) infusion via an infusion pump. Hospitalization is recommended for the first 9 days of the first cycle and the first 2 days of the second cycle. Supervision by a health care professional or hospitalization is recommended for all subsequent cycle starts and re-initiation.^12,13

Erwinaze (asparaginase Erwinia chrysanthemi; EUSA Pharma) received an expanded indication on December 19, 2014, for IV infusion. Erwinaze was first approved for intramuscular injection on November 28, 2011, to treat patients with ALL who had developed an allergy (hypersensitivity) to E coli—derived asparaginase and pegapargase chemotherapy drugs used to treat ALL. Erwinaze can now be administered intravenously or intramuscularly. Erwinaze is supplied as 10,000 IU/vial of lyophilized powder to be reconstituted for injection. The dosage is dependent upon the patient’s body weight, specific condition being treated, and response to therapy.^14,15

Jakafi (ruxolitinib; Incyte Corp) received an expanded indication on December 4, 2014, as the first drug approved by the FDA to treat patients with polycythemia vera who have had an inadequate response to or are intolerant to hydroxyurea. Jakafi was originally approved on November 16, 2011, for the treatment of patients with intermediate or high-risk myelofibrosis. Jakafi is available in 5-mg, 10-mg, 15-mg, 20-mg, and 25-mg tablets for oral use. For myelofibrosis, the starting dose of Jakafi is based on patient’s baseline platelet count, and it is important to monitor complete blood counts every 2 to 4 weeks until doses are stabilized, and then as clinically indicated. The starting dose for polycythemia vera is 10 mg twice daily.^16,17

Oncology—Gastrointestinal

Cyramza (ramucirumab; Eli Lilly and Company) was originally approved on April 21, 2014, as a single agent to treat patients with advanced stomach cancer or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior fluoropyrimidine or platinum-containing chemotherapy. On November 5, 2014, it received an expanded indication to treat patients with advanced gastric or GEJ adenocarcinoma in combination with paclitaxel. Furthermore, on December 12, 2014, it was approved for treatment of metastatic non-small cell lung cancer (NSCLC) with disease progression on or after platinum-based chemotherapy in combination with docetaxel. Cyramza is administered as an IV infusion over 60 minutes. It is available in 100-mg/10 mL or 500-mg/50 mL single-dose vials. The recommended dosage in gastric cancer as a single agent or in combination with weekly paclitaxel is 8 mg/kg every 2 weeks, and in NSCLC the recommended dose is 10 mg/kg intravenously on day 1 of a 21-day cycle prior to docetaxel infusion.^18-21

Somatuline Depot Injection (lanreotide acetate; Ipsen Pharma) received an expanded indication on December 16, 2014, to improve progression-free survival in patients with unresectable, well, or moderately differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors. The recommended dosage is 120 mg administered by deep subcutaneous (SC) injection every 28 days, and treatment should continue until disease progression or unacceptable toxicity. Somatuline Depot is a man-made protein that is similar to a hormone in the body called somatostatin. Somatuline Depot was originally approved on August 31, 2007, for treatment of acromegaly. Somatuline Depot is available as 60-mg/0.2 mL, 90-mg/0.3 mL, and 120-mg/0.5 mL single-use prefilled syringes.^22,23

Oncology—Melanoma

Mekinist (trametinib; GlaxoSmithKline) received an expanded indication on January 9, 2014, for combination use with Tafinlar (dabrafeneb) to treat patients with unresectable melanoma or metastatic melanoma with BRAF V600E or V600K mutations. Mekinist was originally approved on May 29, 2013, as a single-agent, oral treatment for unresectable or metastatic melanoma in adult patients with BRAF V600E or V600K mutations. Mekinist is not indicated for the treatment of patients who have received a prior BRAF inhibitor. Mekinist acts as a reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2 activation and of MEK1 and MEK2 kinase activity. The recommended dosage regimens of Mekinist are 2 mg orally once daily as a single agent or in combination with dabrafenib 150 mg orally twice daily. Mekinist should be taken at least 1 hour before or at least 2 hours after a meal. It must be dispensed and stored in its original bottle.^24,25

Keytruda (pembrolizumab; Merck & Co, Inc) was approved on September 4, 2014, as the first anti-programmed death receptor-1 (PD-1) therapy for the treatment of patients with unresectable or metastatic melanoma and disease progression following Yervoy (ipilimumab) and, if BRAF V600 mutation positive, a BRAF inhibitor. Keytruda is available as 50 mg of lyophilized powder in a single-use vial for reconstitution. The recommended dose is 2 mg/kg as an IV infusion over 30 minutes every 3 weeks.^26,27

Opdivo (nivolumab; Bristol-Myers Squibb) was approved on December 22, 2014, for the treatment of advanced melanoma. Opdivo is a PD-1 blocking antibody indicated for patients who have been previously treated with Yervoy (ipilimu-mab) and, if BRAF V600 mutation positive, a BRAF inhibitor. It is available as a 40-mg/4 mL and 100-mg/10 mL solution for injection in a single-use vial. The recommended dose is 3 mg/kg as an IV infusion over 60 minutes every 2 weeks.^28,29

Oncology—Lung Cancer

Zykadia (ceritinib; Novartis Pharma-ceuticals Corporation) was approved on April 29, 2014, for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic NSCLC. Zykadia is an ALK tyrosine kinase inhibitor that blocks proteins that promote the development of cancerous cells. It is intended for patients with metastatic ALK-positive NSCLC who have been previously treated with crizotinib, the only other approved ALK tyrosine kinase inhibitor. Zykadia is the fourth drug with breakthrough therapy designation that has received FDA approval. Zykadia is available in 150-mg capsules, and the recommended dose is 750 mg orally once daily on an empty stomach.^30,31

Oncology—Ovarian Cancer

Lynparza (olaparib; AstraZeneca) was approved on December 19, 2014, as the first oral poly ADP ribose polymerase inhibitor for monotherapy in patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian cancer who have been treated with 3 or more prior lines of chemotherapy. Lynparza is available in a 50-mg capsule and the recommended dosage is 400 mg orally twice daily. Patients should not take Lynparza with grapefruit or Seville oranges.^32,33

Other Oncology and Oncology Support

Avastin (bevacizumab; Genentech Inc) received an expanded indication on August 14, 2014, for the treatment of patients with persistent, recurrent, or late-stage (metastatic) cervical cancer, and again on November 14, 2014, for the treatment of women with platinum-resistant, recurrent ovarian cancer in combination with chemotherapy. Avastin was originally approved on February 26, 2004, for the treatment of metastatic colorectal cancer and has been approved for the treatment of several types of cancer since then, including non-squamous NSCLC; metastatic renal cell carcinoma; persistent, recurrent, or metastatic cervical cancer; recurrent glioblastoma; and platinum-resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. Avastin is a vascular endothelial growth factor-specific angiogenesis inhibitor available in a 100-mg/4 mL or 400-mg/16 mL single-use vial. For details regarding dosage and administration, please refer to the product prescribing information.^34-36

On December 8, 2014, Xgeva (denosumab; Amgen) obtained an expanded indication for the treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy. Xgeva was originally approved on November 19, 2010, for the prevention of skeletal-related events in patients with bone metastases from solid tumors and has also been approved for the treatment of adults and some adolescents with giant cell tumor of the bone, a rare and usually non-cancerous tumor. Xgeva is not indicated for the prevention of skeletal-related events in patients with multiple myeloma. Xgeva is a Rank ligand inhibitor available as a 120-mg/1.7 mL solution in a single-use vial. It is to be administered subcutaneously into the upper arm, upper thigh, or abdomen. The recommended dosage for Xgeva in hypercalcemia of malignancy is 120 mg every 4 weeks with additional 120-mg doses on days 8 and 15 of the first month of therapy.^37,38

On December 23, 2014, Granix (tbo-filgrastim; Teva) injection received an approval from the FDA for self-administration by patients and caregivers. The approval of this additional administration option will allow physicians to have the flexibility to prescribe Granix for either in-office or at-home use. Granix is a leukocyte growth factor indicated for reduction in the duration of severe neutropenia in patients with non-myeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of febrile neutropenia. It is available in a 300-mcg/0.5 mL or 480-mcg/0.8 mL solution in single-use prefilled syringes for SC use. Granix was originally approved on August 29, 2012, for the treatment of severe neutropenia in certain cancer patients. The recommended dose is 5 mcg/kg per day; the first dose should be administered no earlier than 24 hours following myelosuppressive chemotherapy. Granix should not be administered within 24 hours prior to chemotherapy.^39,40

Bleeding Disorders

Alprolix (coagulation factor IX [recombinant]; Biogen Idec, Inc) was approved on March 28, 2014, for treatment in adults and children with hemophilia B for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis. Alprolix is the first hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. Alprolix is a recombinant DNA—derived coagulation factor IX concentrate. It is to be administered intravenously within 3 hours of reconstitution and is provided in the form of a kit for reconstitution. Alprolix is available as a lyophilized powder in single-use vials containing nominally 500, 1000, 2000, or 3000 IU. Refer to product prescribing information for details regarding dosage and administration.^41,42

Eloctate (antihemophilic factor [Recombinant], fc fusion protein; Biogen Idec, Inc) was approved on June 6, 2014, for treatment in adults and children with hemophilia A for control and prevention of bleeding episodes, perioperative management (surgical prophylaxis), and routine prophylaxis. Eloctate is the first hemophilia A treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding due to its prolonged half-life. Eloctate is a recombinant DNA—derived anti-hemophilic factor available as a lyophilized powder in single-use vials containing nominally 250, 500, 750, 1000, 1500, 2000, or 3000 IU of factor VIII potency. Refer to product prescribing information for details regarding dosage and administration.^43,44

Obizur (antihemophilic factor [recombinant], porcine factor; Baxter Inc) was approved on October 24, 2014, for the treatment of bleeding episodes in adults with acquired hemophilia A (acquired Factor VIII deficiency). Obizur is available as lyophilized powder for solution in single-use vials containing 500 units per vial. The initial dose of Obizur is 200 units/kg administered intravenously after reconstitution. The dose titration and frequency of administration is based on factor VIII recovery levels and individual clinical response. Obizur is not indicated for the treatment of congenital hemophilia A or von Willebrand disease. The safety and efficacy of Obizur has not been established in patients with a baseline anti-porcine factor VIII inhibitor titer of greater than 20 BU.^45,46

Inflammatory Conditions

Monovisc (sodium hyaluronate; Anika Therapeutics, Inc) was approved on February 25, 2014, as a single-injection supplement to the synovial fluid of an osteoarthritic joint, used to treat pain and improve joint mobility in patients suffering from osteoarthritis (OA) of the knee. Monovisc is indicated for the treatment of pain in OA of the knee in patients who have failed to respond adequately to conservative non-pharmacologic therapy and to first-line analgesics such as acetaminophen. Monovisc is the first FDA-approved single-injection product with sodium hyaluronate from a non-animal source. It is a sterile viscoelastic 4-mL preparation supplied in a disposable glass syringe.^47,48

Otezla (apremilast; Celgene Corp) was initially approved on March 21, 2014, for the treatment of adult patients with active psoriatic arthritis. On September 23, 2014, Otezla received an expanded indication for the treatment of patients with moderate to severe plaque psoriasis for whom phototherapy or systemic therapy is appropriate. Otezla is an inhibitor of phosphodiesterase 4 (PDE4). It is available in 10-mg, 20-mg, and 30-mg tablets. The recommended initial dosage of Otezla is based on a titration regimen from days 1 to 5, as detailed in the product prescribing information. Following the 5-day titration, the recommended maintenance dose is 30 mg twice daily taken orally starting on day 6. This titration is intended to reduce the gastrointestinal symptoms associated with initial therapy.^49-51

Entyvio (vedolizumab; Takeda Pharmaceuticals America, Inc) was approved on May 20, 2014, to treat adult patients with moderate to severe ulcerative colitis (UC) and moderate to severe Crohn’s disease (CD) when 1 or more standard therapies (corticosteroids, immunomodulators, or tumor necrosis factor blocker medications) have not resulted in an adequate response. Entyvio is an integrin receptor antagonist. It is available as 300 mg of lyophilized powder in a single-use 20-mL vial. Entyvio must be reconstituted and diluted prior to administration. The recommended dosage in UC and CD is 300 mg infused intravenously over approximately 30 minutes at 0, 2, and 6 weeks, then every 8 weeks thereafter.^52,53

Rasuvo (methotrexate; Medac Pharma, Inc) was approved on July 11, 2014, as an SC, ready-to-use autopen formulation of methotrexate for the treatment of patients with severe, active rheumatoid arthritis (RA) and polyarticular juvenile idiopathic arthritis, who are intolerant of or have had an inadequate response to first-line therapy; and, for symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy. Rasuvo is not indicated for the treatment of neoplastic diseases. Rasuvo is for once-weekly SC use only and is available as a single-dose, manually triggered auto-injector delivering methotrexate in the following dosage strengths: 7.5 mg, 10 mg, 12.5 mg, 15 mg, 17.5 mg, 20 mg, 22.5 mg, 25 mg, 27.5 mg, and 30 mg. Refer to product prescribing information for details regarding dosage regimen.^54,55

On September 25, 2014, Humira (adalimumab; AbbVie) received an expanded indication for the treatment of pediatric patients 6 years and older with Crohn’s disease who have had an inadequate response to corticosteroids or immunomodulators. Humira was originally approved on December 31, 2002, for the treatment of RA and has since received indications for juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn’s disease, UC, and plaque psoriasis. Humira is available in a 40-mg/0.8 mL single-use prefilled pen; 40-mg/0.8 mL, 20-mg/0.4 mL, and 10-mg/0.2 mL single-use prefilled glass syringe; and in a 40-mg/0.8 mL single-use glass vial for institutional use only. Refer to the product prescribing information for dosing and administration details.^56,57

Cystic Fibrosis

Kitabis Pak (tobramycin; PulmoFlow, Inc) was approved on December 2, 2014. Kitabis Pak contains tobramycin, an aminoglycoside inhalation solution, co-packaged with a Pari LC Plus Reusable Nebulizer for the management of cystic fibrosis (CF) in adults and pediatric patients 6 years and older with Pseudomonas aeruginosa. This is the first nebulized drug and device combination to receive FDA approval for patients with CF. The medication is to be administered as 1 single-use ampule (300 mg/5 mL) twice a day by oral inhalation in alternating periods of 28 days on drug, followed by 28 days off drug.^58,59

Kalydeco (ivacaftor; Vertex Pharmaceuticals) received an expanded indication on December 29, 2014, for treatment in patients with CF 6 years and older who have the R117H mutation in the CF transmembrane conductance regulator (CFTR) gene. Kalydeco was originally approved on January 31, 2012, for the treatment of CF in patients 6 years and older who have the specific G551D mutation in the CFTR gene. Kalydeco is a CFTR potentiator indicated for the treatment of CF in patients 6 years and older who have 1 of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. It is available in 150-mg tablets, and the recommended dosage in adults and pediatric patients 6 years and older is one 150-mg tablet taken orally every 12 hours with fat-containing food.^60,61

Pulmonary Fibrosis

Esbriet (pirfenidone; InterMune, Inc) was approved on October 15, 2014, for treatment of idiopathic pulmonary fibrosis (IPF). Esbriet should be taken with food to minimize nausea and dizziness. Esbriet is available in a 267-mg capsule formulation and the recommended dose is 801 mg (2 capsules) 3 times daily with food. Upon initiation of treatment, the daily dosage should be titrated to the full dosage of 9 capsules per day over a 14-day period as detailed in the product prescribing information.^62,63

On October 15, 2014, Ofev (nintedanib; Boehringer Ingelheim Pharmaceuticals, Inc) was approved for the treatment of IPF. Ofev is a small molecule tyrosine kinase inhibitor available in 150-mg and 100-mg capsules. The recommended dose is 150 mg twice daily taken approximately 12 hours apart with food.^64,65

HIV

Triumeq (abacavir 600 mg, dolutegravir 50 mg, and lamivudine 300 mg; ViiV Healthcare) was approved on August 22, 2014, for the treatment of HIV-1 infection. Triumeq is a single-tablet regimen that combines the integrase strand transfer inhibitor dolutegravir, with the nucleoside reverse transcriptase inhibitors abacavir and lamivudine. The dosage in adults is 1 tablet daily, and the medication may be taken with or without food. Since the product contains abacavir, patients should be tested prior to therapy to determine if they carry the HLA-B*5701 allele as those patients are at higher risk of experiencing a hypersensitivity reaction to abacavir.^66,67

Vitekta (elvitegravir; Gilead Sciences Inc) was approved on September 24, 2014, to be used as part of a regimen to treat HIV-1 infection in treatment-experienced adults. Vitekta is an HIV integrase strand transfer inhibitor that works by interfering with 1 of the enzymes that HIV needs to multiply. It is to be used in combination with an HIV protease inhibitor coadministered with ritonavir and with other antiretroviral drugs. Vitekta is one of the components in the combination HIV tablet Stribild, which was approved by the FDA in August 2012. Vitekta is available in 85-mg and 150-mg tablets and is administered once daily with food.^68,69

Tybost (cobicistat; Gilead Sciences Inc) was approved by the FDA on September 24, 2014, for use in combination with Reyataz (atazanavir) or Prezista (darunavir) for the treatment of HIV-1 infection. Tybost is a pharmacokinetic enhancer that works by inhibiting the enzyme (CYP3A) that metabolizes atazanavir and darunavir. It increases the systemic exposure of these drugs and prolongs their effect. It is also 1 of the components in the combination HIV tablet Stribild. Tybost is available in 150 mg tablets and is administered once daily in combination with the protease inhibitors atazanavir or darunavir.^70,71

Hepatitis C

On October 10, 2014, Harvoni (ledipasvir and sofosbuvir; Gilead Sciences, Inc) was approved as the first combination pill to treat chronic hepatitis C virus (HCV) genotype 1 infection in patients 18 years and older. Harvoni contains 90 mg of ledipasvir (an HCV NS5A inhibitor) and 400 mg of sofosbuvir (an HCV nucleotide analog NS5B polymerase inhibitor) in a single tablet. The recommended dosage of Harvoni is 1 tablet taken orally once daily with or without food, although the treatment duration depends on whether the patient is treatment naïve or treatment experienced and whether or not the patient has cirrhosis. Harvoni contains sofosbuvir, which is the active agent in Sovaldi, originally approved by the FDA in December 2013.^72,73

Olysio (simeprevir; Janssen) received an expanded indication on November 5, 2014, to be used in combination with Sovaldi (sofosbuvir), for genotype 1 chronic HCV infection in adult patients. The recommended treatment duration of Olysio when used in conjunction with sofosbuvir is 12 weeks for patients without cirrhosis who are treatment naïve or treatment experienced, or 24 weeks for patients with cirrhosis who are treatment naïve or treatment experienced. Olysio, an HCV NS3/4A protease inhibitor, was originally approved on November 22, 2013 for the treatment of chronic HCV infection as a component of a combination antiviral treatment regimen. Screening patients with HCV genotype 1a infection for the presence of virus with the NS3 Q80K polymorphism at baseline is strongly recommended, and alternative therapy should be considered for patients infected with HCV genotype 1a containing the Q80K polymorphism. The recommended dosage of Olysio is one 150-mg capsule taken once daily with food.^74,75

Viekira Pak (ombitasvir, paritaprevir, and ritonavir with dasabuvir; AbbVie Inc) was approved on December 19, 2014, to treat patients with genotype 1 chronic HCV infection including those with compensated cirrhosis. Viekira Pak includes ombitasvir, an NS5A inhibitor; paritaprevir, an NS3/4A protease inhibitor; and ritonavir, a CYP3A inhibitor, and is co-packaged with dasabuvir, a non-nucleoside NS5B palm polymerase inhibitor. Viekira Pak treatment duration and whether or not it should be given in conjunction with ribavirin is dependant upon the patient’s genotype and whether or not they have cirrhosis. Viekira Pak is taken twice daily with a meal. Each Viekira Pak is dispensed in a monthly carton for a total of 28 days of therapy. Each monthly carton contains 4 weekly cartons and each weekly carton contains 7 daily dose packs.^76,77

Multiple Sclerosis

Copaxone (glatiramer acetate injection; Teva Pharmaceutical Industries Ltd) received a new dosage form approval on January 28, 2014, for 3-times-a-week dosing of Copaxone 40 mg/mL for multiple sclerosis (MS). This new formulation allows for a less frequent dosing regimen to be administered subcutaneously for patients with relapsing forms of MS. It was originally approved on December 23, 1996, for the treatment of patients with relapsing forms of MS. With this approval, Copaxone is now available in both a 20-mg/ml and a 40-mg/ml single-dose prefilled syringe. Copaxone is to be administered as an SC injection at 20 mg/ml per day or 40 mg/ml 3 times a week.^78-80

On August 15, 2014, Plegridy (peginterferon beta-1a; Biogen Idec) was approved for the treatment of patients with relapsing forms of MS. It is the only pegylated beta interferon approved for use in MS. The dose can be administered subcutaneously with a prefilled pen (a ready-to-use auto-injector) or a prefilled syringe. The recommended dose of Plegridy is 125 mcg injected subcutaneously every 14 days. Patients are started at 63 mcg on day 1, increased to 94 mcg on day 15, and then further increased to 125 mcg on day 29 and onward. Starter packs are available for both the prefilled pen and the prefilled syringes to accommodate the titration.^81,82

Lemtrada (alemtuzumab; Genzyme) was approved on November 14, 2014, for the treatment of patients with relapsing forms of MS. Lemtrada is a CD52-directed cytolytic monoclonal antibody and is only available through a restricted distribution program, the Lemtrada REMS (Risk Evaluation and Mitigation Strategy) Program. Lemtrada is to be administered by IV infusion over 4 hours for 2 treatment courses. The first treatment course is administered at a dose of 12 mg/day via IV infusion on 5 consecutive days. The second course is administered at a dose of 12 mg/day on 3 consecutive days 12 months after the first treatment course.^83,84

Angioedema

Kalbitor (ecallantide; Dyax Corp) received an expanded indication on April 3, 2014, for the treatment of acute hereditary angioedema (HAE) attacks in patients 12 years and older. Kalbitor is a peptide inhibitor of plasma kallikrein which was initially approved on December 2, 2009, for the treatment of acute attacks of hereditary angioedema in patients 16 years and older. Kalbitor is supplied as a single-use glass vial containing 10 mg/mL of solution for injection. The recommended dosage is 30 mg (3 mL), administered subcutaneously in three 10-mg (1 mL) injections. If an attack persists, an additional dose of 30 mg may be administered within a 24-hour period.^85,86

On July 17, 2014, Ruconest (C1 esterase inhibitor [recombinant]; Pharming Group NV) was approved as the first recombinant C1-esterase inhibitor product for the treatment of acute attacks in adult and adolescent patients with HAE. Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified (transgenic) rabbits and is intended to restore the level of functional C1 esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling. However, its effectiveness has not been established in HAE patients with laryngeal attacks. Ruconest is available as a lyophilized powder for reconstitution in a single-use vial containing 2100 IU. The recommended dose is based on the patient’s body weight with complete details found in the product prescribing information.^87,88

Other Specialty

Hetlioz (tasimelteon; Vanda Pharma-ceuticals Inc) was approved on January 31, 2014, for the treatment of non-24-hour sleep-wake disorder, which occurs predominately in totally blind individuals. It is the first and only FDA-approved treatment for non-24-hour sleep-wake disorder. Hetlioz is a melatonin receptor antagonist and is available in a 20-mg capsule. The recommended dosage is 20 mg per day taken before bedtime, at the same time every night, without food.^89,90

Vimizim (elosulfase alfa; BioMarin Pharmaceutical Inc) was approved on February 14, 2014, as an enzyme replacement therapy for patients with mucopolysaccharidosis (MPS) type IVA (MPS IVA), also called Morquio A syndrome. MPS IVA is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase, which is an enzyme involved in glycosaminoglycan metabolism. Vimizim is available in a 5-mg/5 mL (1 mg/mL) single-use vial that is to be diluted and injected slowly into a vein through an IV infusion in a clinic or in a hospital setting. The recommended dosage is 2 mg per kg of body weight administered once every week as an IV infusion over a minimum of 3.5 to 4.5 hours, based on the infusion volume. Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion.^91,92

Myalept (metreleptin for injection; manufactured by Amylin Pharmaceuticals, LLC, Myalept was acquired by Aeugerion in November 2014) was approved on February 24, 2014, as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy. Myalept is a leptin analog and is supplied as a sterile, white solid lyophilized cake of 11.3 mg of metreleptin per vial to deliver 5 mg/mL when reconstituted. The recommended daily dosage is dependent upon gender and body weight.^93-95

Xolair (omalizumab; Genentech) received an expanded indication on March 21, 2014, for the treatment of chronic idiopathic urticaria (CIU), a form of chronic hives, in patients 12 years and older who remain symptomatic despite treatment with H1-antihistamine therapy. Xolair is the first biologic medicine and first medicine approved by the FDA for CIU since non-sedating H1-antihistamines. Xolair is available as an SC injection that is to be administered by a health care provider, in a health care setting. The recommended dose for CIU is 150 mg or 300 mg subcutaneously every 4 weeks. Dosing in CIU is not dependent on serum IgE level or body weight. Xolair is an anti-IgE antibody that was initially approved on June 23, 2003, for the treatment of moderate to severe persistent asthma in patients with a positive skin test or in vitro reactivity to a perennial aeroallergen whose symptoms were not adequately controlled with inhaled corticosteroids.^96,97

On July 29, 2014, Eylea received an expanded indication for diabetic macular edema (DME). Eylea is a vascular endothelial growth factor inhibitor formulated as an injection for the eye. Furthermore, on October 6, 2014, Eylea received an additional approval by the FDA for the treatment of patients with macular edema following central retinal vein occlusion. Eylea was originally approved on November 18, 2011, to treat patients with wet (neovascular) age-related macular degeneration, a leading cause of vision loss and blindness in Americans 60 years and older. The recommended dosage of Eylea in patients with DME is 2 mg every 2 months (8 weeks) after 5 initial monthly injections. Eylea may be dosed as frequently as 2 mg every 4 weeks; however, no additional efficacy has been demonstrated with dosing Eylea every 4 weeks compared to every 8 weeks. The recommended dose of Eylea in patients with retinal vein occlusion is 2 mg (0.05 mL) administered by intravitreal injection once every 4 weeks (monthly).^98-100

Lumizyme (alglucosidase alfa; Genzyme) received an expanded indication on August 1, 2014, for the treatment of patients with infantile-onset Pompe disease, including patients who are younger than 8 years. Lumizyme is a lysosomal glycogen-specific enzyme which was first approved by the FDA on May 5, 2010, with a REMS program to restrict its use to treatment of patients with late (non-infantile) onset Pompe disease for patients 8 years and older. The new expanded approval provides access to Lumizyme for all Pompe disease patients, regardless of their age. Health care professionals, health care facilities, and patients will no longer be required to enroll in the Lumizyme REMS program (Lumizyme ACE Program) to be able to prescribe, dispense, or receive Lumizyme. It is available as 50 mg of lyophilized powder in a single-use vial for reconstitution with a recommended dosage of 20 mg/kg body weight administered every 2 weeks as an IV infusion.^101,102

Cerdelga (eliglustat; Genzyme) was approved on August 19, 2014, for the long-term treatment of adult patients with type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers as detected by an FDA-cleared test. Patients who are ultra-rapid CYP2D6 metabolizers may not achieve adequate concentrations of Cerdelga to achieve a therapeutic effect and a specific dosage cannot be recommended for those patients whose CYP2D6 genotype cannot be determined (indeterminate CYP2D6 metabolizers). Cerdelga is a glucosylceramide synthase inhibitor available in an 84-mg capsule formulation. The recommended dosage is based upon an FDA-cleared test for determining CYP2D6 genotype, with extensive metabolizers and intermediate metabolizers receiving 84 mg orally twice daily and poor metabolizers receiving 84 mg orally once daily. Patients should be advised to avoid drinking grapefruit juice or eating grapefruit products while on therapy.^103,104

Promacta (eltrombopag; GlaxoSmithKline) received an expanded indication on August 26, 2014, for treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy. Promacta is an oral thrombopoietin receptor agonist which works by helping to induce proliferation and differentiation of bone marrow stem cells to increase production of blood cells. Promacta was first approved, on November 21, 2008, for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy, and is also approved for the treatment of thrombocytopenia in patients with chronic HCV to allow the initiation and maintenance of interferon-based therapy. Promacta is available in 12.5-mg, 25-mg, 50-mg, 75-mg, and 100-mg tablets. Please see product prescribing information for complete dosing and administration.^105,106

On December 16, 2014, Signifor LAR (pasireotide; Novartis) received FDA approval for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option. Signifor LAR is a somatostatin analog which is available as powder for reconstitution in a 20-mg, 40-mg, and 60-mg vial. It is important to evaluate the patient’s fasting plasma glucose, hemoglobin A1C, liver enzyme tests, electrocardiogram, serum magnesium, and serum potassium prior to starting treatment, as these measures can have a significant impact on the dosage. The recommended initial dose for Signifor LAR is 40 mg, intramuscularly, every 4 weeks. The dose can be adjusted based upon the patient’s biochemical and tolerability response.^107,108 SPT

The above information is a selective summary of publicly available information and is accurate as of the date of writing. Please consult the sources for complete reference information. The views expressed in this article are those of the authors alone and not of Managed Health Care Associates, Inc.

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FDA approves Gilead’s Vitekta (elvitegravir) to treat HIV. AIDSmeds website; September 25, 2014. www.aidsmeds.com/articles/Vitekta_elvitegravir_FDA_1667_26237.shtml. Accessed January 16, 2015.
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FDA approves first combination pill to treat hepatitis C [press release]. FDA website; October 10, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm418365.htm. Accessed January 16, 2015.
Harvoni [prescribing information]. Gilead Sciences; October 2014. www.gilead.com/~/media/Files/pdfs/medicines/liver-disease/harvoni/harvoni_pi.pdf. Accessed January 16, 2015.
Olysio (simeprevir) gains additional FDA approval as once-daily, all-oral interferon- and ribavirin-free treatment option in combination with sofosbuvir for adults with genotype 1 chronic hepatitis C infection [press release]. Johnson & Johnson; November 5, 2014. www.investor.jnj.com/releasedetail.cfm?ReleaseID=881192. Accessed January 16, 2015.
Olysio [prescribing information]. Janssen; November 2013. www.olysio.com/shared/product/olysio/prescribing-information.pdf. Accessed January 16, 2015.
FDA approves Viekira Pak to treat hepatitis C [press release]. FDA website; December 19, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm427530.htm. Accessed January 16, 2015.
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Teva announces US FDA approval of three-times-a-week Copaxone (glatiramer acetate injection) 40 mg/mL [press release]. Teva Pharmaceuticals; January 28, 2014. www.tevapharm.com/Media/News/Pages/2014/1894510.aspx. Accessed January 16, 2015.
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FDA approves rare product to treat genetic disease. FDA website; July 17, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm405526.htm. Accessed January 16, 2015.
Ruconest [prescribing information]. Pharming Group NV; July 17, 2014. https://81b77e9a9bc9711e90b1-f416dd7f832b6e1ad8c969a90667ca99.ssl.cf1.rackcdn.com/shared/pi/ruconest-pi.pdf. Accessed January 16, 2015.
FDA approves Hetlioz: first treatment for non-24 hour sleep-wake disorder [press release]. FDA website; January 31, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm384092.htm. Accessed January 16, 2015.
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FDA approves Myalept to treat rare metabolic disease [press release]. FDA website; February 24, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm387060.htm. Accessed January 16, 2015.
Myalept [prescribing information]. Amylin Pharmaceuticals, LLC; February 2014. www.azpicentral.com/myalept/pi_myalept.pdf#page=1. Accessed January 16, 2015.
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New pediatric labeling information database—detail. FDA website; March 21, 2014. www.accessdata.fda.gov/scripts/sda/sdDetailNavigation.cfm?sd=labelingdatabase&id=0503C379A4B4060BE053554DA8C02A86&rownum=16. Accessed January 16, 2015.
Xolair [prescribing information]. Genentech; June 2003. www.gene.com/download/pdf/xolair_prescribing.pdf. Accessed January 16, 2015.
Eylea (aflibercept) injection receives FDA approval for the treatment of diabetic macular edema (DME) [press release]. Regeneron Pharmaceuticals; July 29, 2014. http://files.shareholder.com/downloads/REGN/3851137698x0x772028/698A12F7-A8DC-4F89-B9E2-F8134AE6F3DE/REGN_News_2014_7_29_General_Releases.pdf. Accessed January 16, 2015.
Eylea (aflibercept) injection receives FDA approval for macular edema following retinal vein occlusion (RVO) [press release]. Regeneron Pharmaceuticals; October 6, 2014. http://files.shareholder.com/downloads/REGN/3851137698x0x785011/289C9A9E-A261-4750-BBEB-6417A3510B5E/REGN_News_2014_10_6_General_Releases.pdf. Accessed January 16, 2015.
Eylea [prescribing information]. Regeneron Pharmaceuticals; November 2011. www.regeneron.com/Eylea/eylea-fpi.pdf. Accessed January 16, 2015.
FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements [press release]. FDA website; August 1, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407563.htm. Accessed January 16, 2015.
Lumizyme [prescribing information]. Genzyme; May 2010. www.lumizyme.com/~/media/LumizymeUS/Files/lumizyme_prescribing_information.pdf. Accessed January 16, 2015.
FDA approves new drug to treat a form of Gaucher disease [press release]. FDA website; August 19, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm410585.htm. Accessed January 16, 2015.
Cerdelga [prescribing information]. Genzyme; August 2014. www.cerdelga.com/pdf/cerdelga_prescribing_information.pdf. Accessed January 16, 2015.
GSK receives FDA approval of an additional Promacta (eltrombopag) indication for use in patients with severe aplastic anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST) [press release]. GlaxoSmith Kline; August 26, 2014. www.gsk.com/en-gb/media/press-releases/2014/gsks-promacta-eltrombopag-receives-fda-approval-of-an-additional-indication/. Accessed January 16, 2015.
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Novartis gains FDA approval for Signifor LAR to treat patients with acromegaly, a rare and life-threatening hormonal disorder [press release]. Novartis Pharmaceuticals; December 16, 2015. www.novartis.com/newsroom/media-releases/en/2014/1880449.shtml. Accessed January 16, 2015.
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About the Author

Stacey Ness, PharmD, RPh, CSP, MSCS, AAHIVP, has worked in both national specialty pharmacy and payer organizations and has experience in clinical management, adherence and persistency programs, as well as chronic disease cost optimization strategies. Dr. Ness is active in the Consortium of Multiple Sclerosis Centers, Academy of Managed Care Pharmacy, National Home Infusion Association, National Association of Specialty Pharmacy, Specialty Pharmacy Certification Board, and Hematology and Oncology Pharmacy Association, and has served on the Minnesota Medicaid Drug Formulary Committee since 2008. She is a multiple sclerosis certified specialist, a credentialed HIV Pharmacist, a certified specialty pharmacist, and currently serves as the director of specialty clinical services at Managed Health Care Associates, Inc, a health care services organization based in Florham Park, New Jersey.

Ruby Mhajan, RPh, currently serves as a clinical pharmacist at Managed Health Care Associates, Inc, a health care services organization based in Florham Park, New Jersey. Ruby has over 13 years of exceptional experience as a clinical pharmacist, having worked in both the United States and Canada in various settings including retail, long-term care, home infusion/specialty, and a national outsourcing admixture pharmacy. Most recently, she served as a director of pharmacy for a home infusion/specialty pharmacy in New York.

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