Specialty Drugs: Year in Review 2017 Part I
Specialty pharmaceuticals featured prominently in the actions of the FDA in 2017.
The year 2017 was another in which specialty pharmaceuticals featured prominently in the actions of the FDA. Below is the first installment of a summary of specialty pharmacy—related FDA approvals and expanded indications that took place in 2017. The second installment will include oncology drugs and late-breaking FDA actions. Please consult product prescribing monographs for complete information including dosing regimens.
Bleeding Disorders
On May 31, 2017, the FDA approved Rebinyn (coagulation factor IX [recombinant], glycopegylated) for the treatment of adults and children with hemophilia B for on-demand treatment and control of bleeding episodes and perioperative management of bleeding. Rebinyn is not indicated for routine prophylaxis in the treatment of patients with hemophilia B or immune tolerance induction in patients with hemophilia B. Rebinyn glycopegylated is an extended—half-life recombinant DNA-derived coagulation factor IX concentrate. Rebinyn is available as a lyophilized powder in single-use vials of 500, 1000, and 2000 international units (IU) of factor IX potency per vial with a MixPro prefilled diluent syringe containing histidine solution and a sterile vial adapter for intravenous (IV) infusion. Dosage and duration of treatment with Rebinyn depend on the severity of factor IX deficiency, the location and extent of bleeding, and the patient’s clinical condition, body weight, and recovery of factor IX.1,2
Inflammatory Conditions
On February 15, 2017, Siliq (brodalumab) received FDA approval for treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy and have failed to respond or lost response to other systemic therapies. Siliq is a human interleukin-17 receptor A antagonist that is available in a single prefilled syringe (PFS) containing 210 mg/1.5 mL solution for subcutaneous (SC) injection. The recommended dosage is 210 mg of Siliq by SC injection at weeks 0, 1, and 2 followed by 210 mg every 2 weeks. Treatment beyond 16 weeks in patients who have not achieved an adequate response is not likely to result in greater success. Siliq is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS)—called the Siliq REMS Program—because of the boxed warning of suicidal ideation and behavior associated with the medication.3,4
On March 28, 2017, Dupixent (dupilumab) received FDA approval for the treatment of adult patients with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The FDA granted the application for Dupixent both Priority Review and Breakthrough Therapy designation. Dupixent can be used with or without topical corticosteroids. The interleukin-4 receptor alpha antagonist is available in a solution of 300 mg/2 mL in a single-dose PFS with a needle shield for SC use. The recommended initial dose is 600 mg (two 300-mg injections in different injection sites), followed by 300 mg every other week. The safety and efficacy of Dupixent has not been established in the treatment of asthma.5,6
On March 30, 2017, Humira (adalimumab) received an approved labeling revision to include data regarding moderate-to-severe fingernail psoriasis in the Humira prescribing information for patients with moderate-to-severe chronic plaque psoriasis. Humira, a tumor necrosis factor (TNF) blocker, was originally approved on December 31, 2002, for the treatment of rheumatoid arthritis. It has since received indications for juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease in adult and pediatric patients 6 years and older, adult ulcerative colitis, plaque psoriasis, uveitis, and moderate-to-severe hidradenitis suppurativa.7,8
On April 21, 2017, Renflexis (infliximab-abda) was approved by the FDA as the second available biosimilar to Remicade (infliximab). Renflexis is indicated for the treatment of adult and pediatric Crohn’s disease, ulcerative colitis, rheumatoid arthritis in combination with methotrexate, ankylosing spondylitis, psoriatic arthritis, and plaque psoriasis. The TNF blocker is available in a 100-mg single-dose vial of lyophilized powder of infliximab-abda for a final reconstitution volume of 10 mL. Renflexis is administered via IV infusion over a period of not less than 2 hours.9,10
On May 22, 2017, Kevzara (sarilumab) was approved by the FDA for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to 1 or more disease-modifying antirheumatic drugs (DMARDs). The interleukin-6 receptor antagonist is available as a solution of 150 mg/1.14 mL or 200 mg/1.14 mL in a single-dose PFS for SC administration. The recommended dose of Kevzara is 200 mg once every 2 weeks. Kevzara may be used as monotherapy or in combination with methotrexate or other conventional DMARDs.11,12
On May 22, 2017, Actemra (tocilizumab) received an expanded indication for the treatment of adults with giant cell arteritis. The FDA granted this application Breakthrough Therapy designation and Priority Review. Furthermore, on August 30, 2017, Actemra received an expanded indication for the treatment of adults and pediatric patients 2 years and older with chimeric antigen receptor T cell—induced severe or life-threatening cytokine release syndrome (CRS). The FDA granted Priority Review and Orphan Drug designation to Actemra for this indication. Actemra is an interleukin-6 receptor antagonist that was originally approved in January 2010. Actemra is also indicated in the treatment of adult patients with moderately to severely active rheumatoid arthritis who had an inadequate response to 1 or more DMARDs and patients 2 years and older with active juvenile idiopathic arthritis. Actemra is available in single-use vials of 20 mg/mL for IV administration and a single-use PFS providing 162 mg of Actemra in 0.9 mL for SC administration. The recommended dose of Actemra for adult patients with giant cell arteritis is 162 mg administered once weekly as an SC injection, in combination with a tapering course of glucocorticoids. A dose of 162 mg administered once every other week as a SC injection, in combination with a tapering course of glucocorticoids, may be prescribed based on clinical considerations. The recommended dose of Actemra for CRS is 12 mg/kg IV (<30 kg) or 8 mg/kg (≥30 kg), alone or in combination with corticosteroids, as a 60-minute IV infusion. If no clinical improvement in the signs and symptoms of CRS occurs after the first dose, up to 3 additional doses of Actemra may be administered. The interval between consecutive doses should be at least 8 hours. SC administration is not approved for CRS.13-15
On July 6, 2017, Orencia (abatacept) received an expanded FDA indication for the treatment of active psoriatic arthritis in adult patients by IV or SC administration. Orencia is a selective T-cell co-stimulation modulator that was originally approved in December 2005. Orencia is also indicated for the treatment of moderately to severely active rheumatoid arthritis in adults and juvenile idiopathic arthritis in patients 2 years and older. Orencia may be used as monotherapy or concomitantly with DMARDs other than TNF antagonists. Orencia is available in a single-use vial containing 250 mg lyophilized powder for IV infusion. Orencia is also available in a solution of 50 mg/0.4 mL, 87.5 mg/0.7 mL, and 125 mg/mL in a single-dose PFS and a solution of 125 mg/mL in a single-dose prefilled ClickJect autoinjector for SC use. The recommended dose of Orencia IV for patients with psoriatic arthritis is dependent on the patient’s weight and is administered as a 30-minute IV infusion. Following the initial IV administration, the next IV infusion should be given at 2 and 4 weeks after the first infusion and every 4 weeks thereafter. The recommended dose of Orencia SC for psoriatic arthritis patients is 125 mg once weekly without the need for an IV loading dose.16,17
On July 13, 2017, Tremfya (guselkumab) was approved by the FDA for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Tremfya is an interleukin-23 blocker that is available in a single-dose PFS of 100 mg/mL. The recommended dose of Tremfya is 100 mg SC at week 0 and week 4 and every 8 weeks thereafter.18,19
On July 21, 2017, Benlysta (belimumab) was approved for a new SC formulation for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy. Benlysta is a B lymphocyte stimulator-specific inhibitor that was first approved in March 2011 for the treatment of SLE via IV administration. Benlysta is now available in a single-dose prefilled autoinjector of 200 mg/mL or single-dose PFS for SC administration. The recommended dose of Benlysta for SC administration is 200 mg once weekly.20,21
On August 25, 2017, Cyltezo (adalimumab-adbm) received FDA approval for the treatment of various inflammatory diseases, including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, juvenile idiopathic arthritis, and plaque psoriasis. The TNF blocker is the second available biosimilar to Humira. Cyltezo is available as a single-use glass PFS of 40 mg/0.8 mL.22,23
HIV
On May 30, 2017, Isentress (raltegravir) was approved for a new dosing option. Isentress HD is a 1200-mg once-daily dose administered orally as two 600-mg film-coated tablets, with or without food, in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults and pediatric patients weighing at least 40 kg who are treatment-naïve or whose virus has been suppressed on an initial regimen of Isentress 400 mg administered twice daily. The coadministration of Isentress HD with aluminum- and/or magnesium-containing antacids and calcium carbonate antacids is not recommended. Isentress was originally approved in October 2007 for the treatment of HIV-1 infection in combination with other antiretroviral agents.24,25
Hepatitis C
On April 7, 2017, Harvoni (ledipasvir/sofosbuvir) received an expanded indication for the treatment of chronic hepatitis C virus (HCV) infection in adolescents with genotype 1, 4, 5, or 6 without cirrhosis or with compensated cirrhosis, who are 12 years and older and weigh at least 35 kg. Harvoni was first approved in October 2014 and is also indicated for adults with genotype 1 infection with decompensated cirrhosis, in combination with ribavirin and for adults with genotype 1 or 4 infection who are liver transplant recipients without cirrhosis or with compensated cirrhosis, in combination with ribavirin. Harvoni contains 90 mg of ledipasvir (NS5A inhibitor) and 400 mg of sofosbuvir (nucleotide analog NS5B polymerase inhibitor) in a single tablet. The recommended dose of Harvoni for adults and pediatric patients is 1 tablet orally once daily with or without food. The duration of treatment depends on the patient’s age, weight, genotype, whether the patient is treatment naïve or treatment experienced, and whether the patient is with or without cirrhosis.26,27
On April 7, 2017, Sovaldi (sofosbuvir) received an expanded indication for the treatment of genotype 2 or 3 chronic HCV infection in adolescents without cirrhosis or with compensated cirrhosis who are 12 years and older and weigh at least 35 kg. Sovaldi was first approved in December 2013 for the treatment of adult patients with genotype 1, 2, 3, or 4 chronic HCV infection without cirrhosis or with compensated cirrhosis as a component of a combination antiviral treatment regimen. Sovaldi is a nucleotide analog NS5B polymerase inhibitor available in a 400-mg tablet. The recommended dose for Sovaldi for adult and pediatric patients is one 400-mg tablet taken once daily with or without food. The addition of ribavirin and duration of treatment depend on the patient’s age, weight, genotype, whether the patient is treatment naïve or treatment experienced, and whether the patient is with or without cirrhosis.28,29
On July 18, 2017, Vosevi (sofosbuvir/velpatasvir/voxilaprevir) was approved by the FDA for the treatment of adult patients with chronic HCV infection without cirrhosis or with compensated cirrhosis (Child-Pugh A) who have genotypes 1 to 6 and were previously treated with an HCV regimen containing an NS5A inhibitor or have genotype 1a or 3 infection and were previously treated with an HCV regimen containing sofosbuvir without an NS5A inhibitor. Vosevi is a fixed-dose combination of sofosbuvir, velpatasvir (NS5A inhibitor), and voxilaprevir (NS3/4A protease inhibitor). Vosevi is available in a tablet formulation consisting of 400 mg of sofosbuvir, 100 mg of velpatasvir, and 100 mg of voxilaprevir. The recommended dose of Vosevi is 1 tablet taken orally once daily with food.30,31
On August 1, 2017, Epclusa (sofosbuvir/velpatasvir) received an expanded indication by the FDA for the treatment of adults with chronic HCV infection to include use in patients coinfected with HIV. Epclusa was originally approved in June 2016 as a single agent for the treatment of chronic HCV genotype 1, 2, 3, 4, 5, or 6 infection in adult patients with or without compensated cirrhosis. In patients with decompensated cirrhosis, Epclusa can be used in combination with ribavirin. Epclusa is a fixed-dose combination tablet consisting of 400 mg of sofosbuvir and 100 mg of velpatasvir. The recommended dose of Epclusa is 1 tablet taken orally once daily with or without food. For patients without cirrhosis and patients with compensated cirrhosis (Child-Pugh A), the duration of therapy is 12 weeks, and for patients with decompensated cirrhosis (Child-Pugh B or C), the duration of therapy for Epclusa plus ribavirin is 12 weeks.32,33
On August 3, 2017, Mavyret (glecaprevir/pibrentasvir) was approved by the FDA for the treatment of adults with chronic HCV genotypes 1 through 6 without cirrhosis or with mild cirrhosis, including patients with moderate-to-severe kidney disease and those who are on dialysis. Mavyret is also approved for adult patients with HCV genotype 1 infection who have been previously treated with a regimen containing either an NS5A inhibitor or an NS3/4A protease inhibitor, but not both. Mavyret was granted Priority Review and Breakthrough Therapy designation by the FDA. Mavyret is a fixed-dose combination tablet consisting of 100 mg of glecaprevir (NS3/4A protease inhibitor) and 40 mg of pibrentasvir (NS5A inhibitor). The recommended dose of Mavyret is 3 tablets (glecaprevir 300 mg and pibrentasvir 120 mg) taken orally once daily with food. The duration of treatment can range from 8 to 16 weeks depending on the patient’s treatment history, viral genotype, and cirrhotic status.34,35
Multiple Sclerosis
On March 28, 2017, Ocrevus (ocrelizumab) was approved by the FDA for the treatment of patients with relapsing or primary progressive forms of multiple sclerosis. Ocrevus is a CD20-directed cytolytic antibody that is available in a single-dose vial of 300 mg/10mL (30 mg/mL) for IV use. The recommended initial dose of Ocrevus is 300 mg followed 2 weeks later by a second dose of 300 mg and then a single 600-mg dose every 6 months. Premedication with an IV corticosteroid is recommended prior to each Ocrevus infusion to reduce the frequency and severity of infusion reactions.36,37
Cystic Fibrosis
Kalydeco (ivacaftor) received an expanded approval on May 17, 2017, for the treatment of cystic fibrosis (CF). The approval triples the number of rare gene mutations that Kalydeco can now treat, expanding the indication from the treatment of 10 mutations to 33. Kalydeco, originally approved in January 2012, is a CF transmembrane conductance regulator (CFTR) potentiator, indicated for the treatment of CF in patients 2 years and older who have 1 mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. Kalydeco is available in 150-mg tablets and unit dose packets of 50- and 75-mg oral granules. The recommended dose of Kalydeco in adults and pediatric patients 6 years and older is one 150 mg tablet taken orally every 12 hours with fat-containing food. The dosage for pediatric patients 2 years old to less than 6 years and weighing less than 14 kg is one 50 mg packet given orally every 12 hours; and for pediatric patients 2 years of age to less than 6 years and weighing greater than 14 kg is one 75-mg packet given orally every 12 hours. Each Kalydeco packet is to be mixed with 1 teaspoon (5 mL) of soft food or liquid and given with fat-containing food. Kalydeco is not recommended in patients less than 2 years of age. The dose should be reduced in patients with moderate and severe hepatic impairment or when Kalydeco is coadministered with moderate or strong CYP3A inhibitors.38,39
Other Specialty Approvals
On January 5, 2017, Lucentis (ranibizumab) received an expanded approval for the treatment of patients with myopic choroidal neovascularization (mCNV), a complication of severe nearsightedness that can lead to blindness. Furthermore, on April 17, 2017, Lucentis received an expanded approval for the monthly treatment of all forms of diabetic retinopathy. Lucentis, a vascular endothelial growth factor inhibitor, was originally approved in June 2006 for the treatment of patients with neovascular (wet) age-related macular degeneration. Lucentis is also approved for macular edema following retinal vein occlusion, for diabetic macular edema, and for the treatment of diabetic retinopathy in patients with diabetic macular edema. Lucentis is available in 2 different dosage forms and strengths. It is available in a single-use PFS designed to provide 0.05 mL for intravitreal injections, a solution of 10 mg/mL (Lucentis 0.5 mg), and in single-use glass vials designed to provide 0.05 mL for intravitreal injection, 10 mg/mL (Lucentis 0.5 mg), and 6 mg/mL (Lucentis 0.3 mg). The recommended dose for mCNV is 0.5 mg (0.05 mL) administered by intravitreal injection once per month for up to 3 months with retreatment if necessary. The initial recommended dose of Lucentis in patients with diabetic retinopathy is 0.3 mg (0.05 mL) by intravitreal injection once per month.40-42
On February 7, 2017, Parsabiv (etelcalcetide) was approved for the treatment of secondary hyperparathyroidism in adult patients with chronic kidney disease (CKD) on hemodialysis. Parsabiv has not been studied in adult patients with parathyroid carcinoma, primary hyperparathyroidism, or CKD who are not on hemodialysis and is not recommended for use in these populations. Parsabiv is a calcium-sensing receptor agonist and is available as an IV injection in single-dose vials containing 2.5 mg/0.5 mL, 5 mg/mL, and 10 mg/2 mL. The recommended starting dose for Parsabiv is 5 mg administered by IV bolus injection 3 times per week at the end of hemodialysis treatment. The maintenance dose of Parsabiv is individualized and determined by titration based on parathyroid hormone and corrected serum calcium response. The dose range of Parsabiv is 2.5 to 15 mg 3 times per week.43,44
On February 9, 2017, Emflaza (deflazacort) was approved by the FDA for the treatment of patients 5 years and older with Duchenne muscular dystrophy. Emflaza was granted Fast Track designation, Priority Review, and Orphan Drug designation by the FDA. Emflaza is a corticosteroid that is available in 6-mg, 18-mg, 30-mg, and 36-mg tablets or in an oral suspension containing 22.75 mg/mL. The recommended dose of Emflaza is a once-daily dosage of approximately 0.9 mg/kg/day administered orally with or without food. Emflaza should be discontinued gradually when administered for more than a few days.45,46
!--page> On February 28, 2017, Xermelo (telotristat ethyl) was approved by the FDA for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy. Xermelo is a tryptophan hydroxylase inhibitor that is available in a 250-mg tablet for oral use and is dispensed in a monthly case. The recommended dose of Xermelo in adult patients is 250 mg 3 times daily with food for patients whose diarrhea is inadequately controlled by an SSA therapy.47,48
On April 3, 2017, Austedo (deutetrabenazine) was approved for the treatment of chorea associated with Huntington disease and was granted Orphan Drug designation by the FDA. Furthermore, on August 30, 2017, Austedo received an expanded indication for the treatment of tardive dyskinesia in adults. Austedo, a vesicular monoamine transporter 2 (VMAT2) inhibitor, is available in 6-mg, 8-mg, and 12-mg tablets for oral use. The dose of Austedo is determined individually for each patient based on reduction of chorea or tardive dyskinesia and tolerability. The initial recommended dose of Austedo for patients who are not being switched from tetrabenazine (a related VMAT2 inhibitor) is 6 mg once daily with food for Huntington disease and 12 mg per day (6 mg twice daily) with food for tardive dyskinesia. The dose of Austedo may be increased at weekly intervals in increments of 6 mg per day to a maximum recommended daily dose of 48 mg.49-51
On April 25, 2017, Praluent (alirocumab) received an expanded approval of a new supplemental Biologics License Application for a once-monthly (every 4 weeks) 300-mg dose of Praluent injection as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein cholesterol. Praluent, a proprotein convertase subtilisin kexin type 9 inhibitor antibody, was initially approved by the FDA in July 2015. The effect of Praluent on cardiovascular morbidity and mortality has not been determined. Praluent is available in a strength of 75 mg/mL or 150 mg/mL in either a single-dose prefilled pen or a single-dose PFS for SC use that patients can self-administer. The recommended starting dose of Praluent is 75 mg SC once every 2 weeks. An alternative starting dosage for patients who prefer less frequent dosing is 300 mg SC once every 4 weeks.52,53
On April 27, 2017, the FDA approved Brineura (cerliponase alfa) for the treatment of slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2, also known as tripeptidyl peptidase 1 deficiency. Brineura was granted Priority Review and Breakthrough Therapy and Orphan Drug designations. Brineura also received a rare pediatric disease priority review voucher by the FDA. Brineura is a hydrolytic lysosomal N-terminal tripeptidyl peptidase. Brineura is available for intraventricular use in a solution of 150 mg/5 mL (30 mg/mL), 2 single-dose vials per carton co-packaged with intraventricular electrolytes injection, 5 mL, in a single-dose vial. The recommended dose of Brineura is 300 mg administered once every other week as an intraventricular infusion followed by infusion of intraventricular electrolytes over approximately 4.5 hours.54,55
On May 5, 2017, Radicava (edaravone) was approved for the treatment of amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease. The FDA granted Radicava Orphan Drug designation. The mechanism by which Radicava exerts its therapeutic effect in patients with ALS is unknown. Radicava is available in a single-dose polypropylene bag of 30 mg/100 mL for infusion. The recommended dose of Radicava is 60 mg administered as an IV infusion over 60 minutes. The initial treatment cycle consists of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing for 10 days of a 14-day period, followed by a 14-day drug-free period.56,57
On June 16, 2017, Dysport (abobotulinumtoxinA) received an expanded approval by the FDA for the treatment of lower limb spasticity in adults, based on its supplemental Biologics License Application. Dysport, an acetylcholine release inhibitor and a neuromuscular blocking agent, was originally approved in April 2009. Dysport is indicated for the treatment of cervical dystonia in adults, glabellar lines in adult patients less than 65 years of age, upper limb spasticity in adults, and lower limb spasticity in pediatric patients 2 years and older. Dysport is supplied as a 300- or 500-unit lyophilized powder in a sterile, single-use glass vial for reconstitution with preservative-free 0.9% sodium chloride injection USP. Dysport is to be injected intramuscularly, and the dose is dependent on the muscles affected, severity of muscle spasticity, prior response, and adverse reaction history following treatment with Dysport or other botulinum toxin A.58,59
On June 22, 2017, Haegarda (C1 esterase inhibitor [human]) SC was approved by the FDA for routine prophylaxis to prevent hereditary angioedema attacks in adolescent and adult patients. Haegarda is a plasma-derived concentrate of C1 esterase inhibitor (human) that received Orphan Drug designation by the FDA. Haegarda is available in single-use vials containing 2000 or 3000 IU of lyophilized powder that is to be reconstituted with sterile water for injection. The recommended dose of Haegarda is 60 IU per kg body weight SC twice weekly (every 3 or 4 days).60,61
On June 30, 2017, Triptodur (triptorelin) received FDA approval for the treatment of pediatric patients 2 years and older with central precocious puberty. Triptodur is a gonadotropin-releasing hormone agonist that is available for administration as an extended-release injectable suspension for intramuscular (IM) use. Triptodur comes in a 22.5-mg single-use kit containing 1 single-dose vial of Triptodur 22.5 mg lyophilized powder cake, 1 sterile glass syringe with 2 mL of sterile water for injection, and sterile needles. The recommended dose for Triptodur is 1 IM injection of 22.5 mg once every 24 weeks administered under the supervision of a physician.62,63
On August 25, 2017, KedRAB (rabies immunoglobulin human) received approval by the FDA for passive, transient postexposure prophylaxis of rabies infection when administered immediately after contact with a rabid or possibly rabid animal. KedRAB should be administered concurrently with a full course of rabies vaccine. KedRAB is a human rabies immunoglobulin that is available in single-use vials containing a ready-to-use solution of 2 mL or 10 mL with a potency of 150 IU/mL. The recommended dose of KedRAB is 20 IU/kg body weight, given as an IM injection at the time of the first vaccine dose. KedRAB and the first dose of rabies vaccine should be given as soon as possible after exposure to prevent lethal harm to the patient.64,65
On September 6, 2017, Tracleer (bosentan) received an expanded indication and approval of a new 32-mg tablet for use in pediatric patients 3 years and older with idiopathic or congenital pulmonary arterial hypertension to improve pulmonary vascular resistance, which is expected to result in an improvement in exercise ability. The 32-mg Tracleer tablet is scored and can be dispersed in a teaspoon of water before oral administration. Tracleer is available only through the Tracleer REMS program. Tracleer is an endothelin receptor antagonist that was originally approved in November 2001. The recommended dose of Tracleer in pediatric patients is dependent on the weight of the child. Tracleer is also available for adult use in 62.5-mg and 125-mg film-coated tablets.66,67
On September 14, 2017, Privigen (immune globulin intravenous [human], 10% liquid) received an expanded indication for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. Privigen maintenance therapy in CIDP has not been studied for periods longer than 6 months. Privigen was originally approved in July 2007 and is also indicated for the treatment of primary humoral immunodeficiency and chronic immune thrombocytopenic purpura in patients 15 and older. Privigen is available in single-use vials containing 10% immunoglobulin G (0.1 g/mL) solution. The available vial sizes and strengths are 5 gm/50 mL, 10 gm/100 mL, 20 gm/200 mL, and 40 gm/400 mL. The loading dose of Privigen for CIDP is 2 g/kg (20 mL/kg) in divided doses over 2 to 5 consecutive days at an initial infusion rate of 0.5 mg/kg/min; the maintenance dose is 1g/kg (10 mL/kg) administered in 1 to 2 infusions on consecutive days every 3 weeks. The maintenance infusion rate can be increased to 8 mg/kg/min as tolerated.68,69
DISCLOSURE
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 are not of Managed Health Care Associates, Inc. All trademarks and registered trademarks are the property of their respective owners.
!--page> References
1. Rebinyn® Prescribing Information, Novo Nordisk, May 2017. Available at: http://www.novo-pi.com/rebinyn.pdf Accessed: June 27, 2017.
2. FDA Approves New Novo Nordisk Treatment for Patients with Hemophilia, Novo Nordisk, May 31, 2017. Available at: http://press.novonordisk-us.com/2017-05-31-FDA-Approves-New-Novo-Nordisk-Treatment-for-Patients-with-Hemophilia Accessed: June 27, 2017.
3. Siliq™ Prescribing Information, Valeant Pharmaceuticals, Inc., February 2017. Available at: https://www.drugs.com/newdrugs/fda-approves-siliq-brodalumab-plaque-psoriasis-4489.html Accessed: March 8, 2017.
4. FDA approves new psoriasis drug, US FDA, February 15, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm541981.htm Accessed: March 8, 2017.
5. Dupixent® Prescribing Information, Sanofi and Regeneron Pharmaceuticals, Inc., March 2017. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761055lbl.pdf Accessed: April 5, 2017.
6. FDA approves new eczema drug Dupixent®, US FDA, March 28, 2017. Available at: https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm549078.htm Accessed: April 5, 2017.
7. Humira® Prescribing Information, AbbVie, May 2017. Available at: http://www.rxabbvie.com/pdf/humira.pdf Accessed: May 20, 2017.
8. U.S. FDA Approves Addition of Moderate to Severe Fingernail Psoriasis Data to AbbVie's Humira® (adalimumab) Prescribing Information, AbbVie, March 30, 2017. Available at: https://news.abbvie.com/news/us-fda-approves-addition-moderate-to-severe-fingernail-psoriasis-data-to-abbvies-humira-adalimumab-prescribing-information.htm Accessed: May 20, 2017.
9. Renflexis™ Prescribing Information, Samsung Bioepis Co. Ltd, April 21, 2017 Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761054lbl.pdf Accessed: April 27, 2017.
10. Merck announces Samsung Bioepis receives approval of Renflexis™ (Infliximab), a Biosimilar of Remicade®, in Korea, Merck, April 21, 2017 Available at: http://www.businesswire.com/news/home/20151207005084/en/Merck-Announces-Samsung-Bioepis-Receives-Approval-RENFLEXIS%E2%84%A2 Accessed: April 27, 2017.
11. Kevzara® Prescribing Information, Sanofi and Regeneron Pharmaceuticals, Inc., May 2017. Available at: http://products.sanofi.us/kevzara/kevzara.pdf Accessed: May 25, 2017.
12. Regeneron and Sanofi announce FDA approval of Kevzara® (sarilumab) for the treatment of moderately to severely active rheumatoid arthritis in adult patients, Regeneron Pharmaceuticals, Inc., May 22, 2017. Available at: http://investor.regeneron.com/releaseDetail.cfm?releaseid=1027419 Accessed: May 25, 2017.
13. Actemra® Prescribing Information, Genentech USA Inc., August 2017. Available at: https://www.gene.com/download/pdf/actemra_prescribing.pdf Accessed: June 22, 2017.
14. FDA approves first drug to specifically treat giant cell arteritis, USA FDA, May 22, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559791.htm Accessed: June 22, 2017.
15. FDA approves Genentech’s Actemra® (tocilizumab) for the treatment of CAR T Cell-Induced Cytokine Release Syndrome, Genentech, August 30, 2017. Available at: https://www.gene.com/media/press-releases/14679/2017-08-30/fda-approves-genentechs-actemra-tocilizu Accessed: August 31, 2017.
16. Orencia® Prescribing Information, Bristol Myers Squibb, July 2017. Available at: http://packageinserts.bms.com/pi/pi_orencia.pdf Accessed: August 30, 2017.
17. Bristol-Myers Squibb’s Orencia® (abatacept) receives FDA approval for treatment of Active Psoriatic Arthritis (PsA) in adults, Bristol Myers Squibb, July 6, 2017. Available at: https://news.bms.com/press-release/rd-news/bristol-myers-squibbs-orencia-abatacept-receives-fda-approval-treatment-active Accessed: August 30, 2017.
18. Tremfya™ Prescribing Information, Janssen Biotech, Inc., July 2017. Available at: https://www.tremfyainfo.com/shared/product/tremfya/prescribing-information.pdf Accessed: August 30, 2017.
19. Janssen announces U.S. FDA approval of Tremfya™ (guselkumab) for the treatment of moderate to severe plaque psoriasis, Janssen Biotech, Inc., July 13, 2017. Available at: https://www.jnj.com/media-center/press-releases/janssen-announces-us-fda-approval-of-tremfya-guselkumab-for-the-treatment-of-moderate-to-severe-plaque-psoriasis Accessed: August 30, 2017.
20. Benlysta® Prescribing Information, Human Genome Sciences, Inc. and GlaxoSmithKline, July 2017. Available at: https://www.gsksource.com/pharma/content/dam/GlaxoSmithKline/US/en/Prescribing_Information/Benlysta/pdf/BENLYSTA-PI-MG-IFU-COMBINED.PDF Accessed: August 30, 2017.
21. GSK receives FDA approval for a new self-injectable formulation of Benlysta® (belimumab) for systemic lupus erythematosus, Glaxo Smith Kline, July 21, 2017. Available at: https://www.gsk.com/en-gb/media/press-releases/gsk-receives-fda-approval-for-a-new-self-injectable-formulation-of-benlysta-belimumab-for-systemic-lupus-erythematosus/ Accessed: August 30, 2017.
22. Boehringer Ingelheim Pharmaceuticals, Inc. receives FDA approval for Cyltezo™ (adalimumab-adbm), a biosimilar to Humira®, for the treatment of multiple chronic inflammatory diseases, Boehringer Ingelheim Pharmaceuticals, Inc., August 25, 2017. Available at: https://www.boehringer-ingelheim.us/press-release/boehringer-ingelheim-pharmaceuticals-inc-receives-fda-approval-cyltezo-adalimumab Accessed: August 31, 2017.
23. Cyltezo™ Prescribing Information, Boehringer Ingelheim Pharmaceuticals, Inc., August 2017. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761058lbl.pdf Accessed: August 31, 2017.
24. Isentress® HD Prescribing Information, Merck & Co., Inc., May 2017. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/022145s036,203045s013,205786s004lbl.pdf June 27, 2017.
25. Merck receives FDA approval of Isentress® HD (raltegravir), a new once-daily option, in combination with other antiretroviral agents, for the treatment of HIV-1 infection in appropriate patients, Merck & Co., May 30, 2017. Available at: http://investors.merck.com/news/press-release-details/2017/Merck-Receives-FDA-Approval-of-ISENTRESS-HD-raltegravir-a-New-Once-Daily-Option-in-Combination-with-Other-Antiretroviral-Agents-for-the-Treatment-of-HIV-1-Infection-in-Appropriate-Patients/default.aspx June 27, 2017.
26. Harvoni® Prescribing Information. Gilead Sciences, April 2017. Available at: http://www.gilead.com/~/media/Files/pdfs/medicines/liver-disease/harvoni/harvoni_pi.pdf Accessed: April 26, 2017.
27. FDA approves two hepatitis C drugs for pediatric patients, US FDA, April 7, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm551407.htm Accessed: April 26, 2017.
28. Sovaldi® Prescribing Information, Gilead Sciences, Inc., April 2017. Available at: https://www.gilead.com/~/media/Files/pdfs/medicines/liver-disease/sovaldi/sovaldi_pi.pdf Accessed: April 26, 2017.
29. FDA approves two hepatitis C drugs for pediatric patients, US FDA, April 7, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm551407.htm Accessed: April 26, 2017.
30. FDA approves Vosevi™ for Hepatitis C, US FDA, July 18, 2017. Available at: https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm567467.htm Accessed: August 30, 2017.
31. Vosevi™ Prescribing Information, Gilead Sciences, Inc., July 2017. Available at: https://www.gilead.com/~/media/files/pdfs/medicines/liver-disease/vosevi/vosevi_pi.pdf?la=en Accessed: August 30, 2017.
32. Epclusa® Prescribing Information, Gilead Sciences, Inc. August 2017. Available at: https://www.gilead.com/~/media/files/pdfs/medicines/liver-disease/epclusa/epclusa_pi.pdf?la=en Accessed: August 31, 2017.
33. U.S. FDA approves expanded labeling for Epclusa® (sofosbuvir/velpatasvir) for the treatment of Chronic Hepatitis C in patients co-infected with HIV, Gilead Sciences, Inc., August 1, 2017. Available at: http://www.gilead.com/news/press-releases/2017/8/us-fda-approves-expanded-labeling-for-epclusa-sofosbuvirvelpatasvir-for-the-treatment-of-chronic-hepatitis-c-in-patients-coinfected-with-hiv Accessed: August 31, 2017.
34. FDA approves Mavyret™ for Hepatitis C, AbbVie, Inc., August 31, 2017. Available at: https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm570038.htm Accessed: August 31, 2017.
35. Mavyret™ Prescribing Information, AbbVie, Inc., August 2017. Available at: http://www.rxabbvie.com/pdf/mavyret_pi.pdf Accessed: August 31, 2017.
36. Ocrevus™ Prescribing Information, Genentech, Inc., March 2017 Available at: https://www.gene.com/download/pdf/ocrevus_prescribing.pdf Accessed: April 25, 2017.
37. FDA approves new drug to treat multiple sclerosis, US FDA, March 28, 2017. Available at: https://www.gene.com/download/pdf/ocrevus_prescribing.pdf Accessed: April 25, 2017.
38. Kalydeco® Prescribing Information, Vertex Pharm, May 2017. Available at: http://pi.vrtx.com/files/uspi_ivacaftor.pdf Accessed: June 22, 2017.
39. FDA expands approved use of Kalydeco® to treat additional mutations of Cystic Fibrosis, US FDA, May 17, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.htm Accessed: June 22, 2017.
40. Lucentis® Prescribing Information, Genentech, April 2017. Available at: https://www.gene.com/download/pdf/lucentis_prescribing.pdf Accessed: April 26, 2017
41. FDA Approves Genentech’s Lucentis® (Ranibizumab Injection) for Myopic Choroidal Neovascularization, Genentech, January 5, 2017. Available at: https://www.gene.com/media/press-releases/14651/2017-01-05/fda-approves-genentechs-lucentis-ranibiz Accessed: March 7, 2017.
42. FDA Approves Genentech’s Lucentis® (Ranibizumab Injection) for Diabetic Retinopathy, the leading cause of blindness among working age adults in the United States, Genentech, Inc., April 17, 2017. Available at: https://www.gene.com/media/press-releases/14661/2017-04-17/fda-approves-genentechs-lucentis-ranibiz Accessed: April 26, 2017.
43. Parsabiv™ Prescribing Information, Amgen, Inc., February 2017. Available at: http://pi.amgen.com/~/media/amgen/repositorysites/pi-amgen-com/parsabiv/parsabiv_pi.ashx Accessed: March 7, 2017.
44. FDA approves Amgen's Parsabiv™ (Etelcalcetide), first new treatment in more than a decade for Secondary Hyperparathyroidism in adult patients on Hemodialysis, Amgen, Inc., February7,2017. Available at: https://www.amgen.com/media/news-releases/2017/02/fda-approves-amgens-parsabiv-etelcalcetide-first-new-treatment-in-more-than-a-decade-for-secondary-hyperparathyroidism-in-adult-patients-on-hemodialysis/ Accessed: March 7, 2017.
45. FDA approves drug to treat Duchenne Muscular Dystrophy, US FDA, February 9, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm Accessed: March 8, 2017.
46. Emflaza™ Prescribing Information, Marathon Pharmaceuticals, LLC., February 2017. Available at: https://emflaza.com/prescribing-info.pdf Accessed: March 8, 2017.
47. Xermelo™ Prescribing Information, Lexicon Pharmaceuticals, Inc., February 2017. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208794s000lbl.pdf Accessed: March 8, 2017.
48. FDA approves Xermelo™ for carcinoid syndrome diarrhea, US FDA, February 28, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm544035.htm Accessed: March 8, 2017.
49. Austedo™ Prescribing Information, Teva Pharmaceutical Industry Ltd., April 2017. Available at: https://www.austedo.com/hd/pi Accessed: May 20, 2017.
50. Teva announces FDA approval of Austedo™ (deutetrabenazine) tablets for the treatment of Chorea associated with Huntington’s Disease., Teva Pharmaceuticals, April 3, 2017. Available at: http://news.tevausa.com/phoenix.zhtml?c=251945&p=irol-newsArticle&ID=2259487 Accessed: May 20, 2017.
51. Teva announces FDA approval of Austedo® (deutetrabenazine) tablets for the treatment of Tardive Dyskinesia in adults, Teva Pharmaceuticals, August 30, 2017. Available at: http://news.tevausa.com/phoenix.zhtml?c=251945&p=irol-newsArticle&ID=2297470 Accessed: May 20, 2017.
52. Praluent® Prescribing Information, Sanofi-Aventis, April 2017. Available at: http://products.sanofi.us/praluent/praluent.pdf Accessed: May 20, 2017.
53. Sanofi and Regeneron announce FDA approval of a new once-monthly dosing option for Praluent® (alirocumab) injection, Sanofi and Regeneron Pharmaceuticals, Inc., April 25, 2017. Available at: http://www.news.sanofi.us/2017-04-25-Sanofi-and-Regeneron-Announce-FDA-Approval-of-a-New-Once-Monthly-Dosing-Option-for-Praluent-R-alirocumab-Injection Accessed: June 21, 2017.
54. FDA approves first treatment for a form of Batten disease, US FDA, April 27, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555613.htm Accessed: June 21, 2017.
55. Brineura™ Prescribing Information, BioMarin Pharmaceuticals Inc., April 2017. Available at: http://brineura.com/downloads/Brineura_PI.pdf Accessed: June 21, 2017.
56. FDA approves drug to treat ALS, US FDA, May 5, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm557102.htm Accessed: June 22, 2017.
57. Radicava™ Prescribing Information, Mitsubishi Tanabe Pharma, May 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm557102.htm Accessed: June 22, 2017.
58. Dysport® Prescribing Information, Ipsen Bio Pharmaceuticals, Inc., June 2017. Available at: https://www.dysport.com/pdfs/Dysport_Full_Prescribing_Information.pdf Accessed: June 30, 2017.
59. Ipsen announces FDA approval of Dysport® (abobotulinumtoxinA) for the treatment of lower limb spasticity in adults, Ipsen Bio Pharmaceuticals, Inc., June 16, 2017. Available at: https://www.ipsen.com/websites/IPSENCOM-PROD/wp-content/uploads/2017/06/16182401/16-06-2017-sNDA-DYSPORT-ALL-US-FINAL-1.pdf Accessed: June 30, 2017.
60. Haegarda® Prescribing Information, US FDA, June 22, 2017. Available at: http://labeling.cslbehring.com/PI/US/HAEGARDA/EN/HAEGARDA-Prescribing-Information.pdf Accessed: June 28, 2017.
61. FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease, US FDA, June 22, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm564332.htm Accessed: June 28, 2017. Arbor Pharmaceuticals, LLC Announces FDA Approval for Triptodur™, Arbor Pharmaceuticals, LLC, June 30, 2017. Available at: http://arborpharma.com/06292017.php Accessed: August 30, 2017.
62. Triptodur™ Prescribing Information, Arbor Pharmaceuticals, LLC, June 2017. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208956s000lbl.pdf Accessed: August 30, 2017.
63. Arbor Pharmaceuticals, LLC Announces FDA Approval for Triptodur™, Arbor Pharmaceuticals, LLC., June 30, 2017. Available at: http://arborpharma.com/06292017.php Accessed: August 30, 2017.
64. Kedrion Biopharma and Kamada receive FDA approval of KedRAB™, Kedrion BioPharma, August 25, 2017. Available at: https://www.kedrion.us/kedrion-biopharma-and-kamada-receive-fda-approval-kedrab%E2%84%A2 Accessed: September 1, 2017.
65. KedRAB™ Rabies Immune Globulin (Human) Prescribing Information, Kedrion BioPharma, August 2017. Available at: http://www.kedrab.com/KEDRAB-prescribing-information.pdf Accessed: September 1, 2017.
66. Tracleer® Prescribing Information, Actelion Pharmaceuticals, September 2017. Available at: http://www.tracleer.com/assets/PDFs/Tracleer_Full_Prescribing_Information.pdf Accessed: September 15, 2017.
67. Actelion receives FDA approval of Tracleer® (bosentan) for use in pediatric patients with pulmonary arterial hypertension, Johnson & Johnson, September 6, 2017. Available at: https://www.jnj.com/media-center/press-releases/actelion-receives-fda-approval-of-tracleer-bosentan-for-use-in-pediatric-patients-with-pulmonary-arterial-hypertension Accessed: September 15, 2017.
68. Privigen® Prescribing Information, CSL Behring, September 2017. Available at: http://labeling.cslbehring.com/PI/US/Privigen/EN/Privigen-Prescribing-Information.pdf Accessed: September 19, 2017.
69. CSL Behring announces FDA approval of Privigen® [Immune Globulin Intravenous (Human), 10% Liquid] for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in adults, CSL Behring, September 14, 2017. Available at: http://www.prnewswire.com/news-releases/csl-behring-announces-fda-approval-of-privigen-immune-globulin-intravenous-human-10-liquid-for-the-treatment-of-chronic-inflammatory-demyelinating-polyneuropathy-cidp-in-adults-300520181.html Accessed: September 19, 2017.
-->
