Specialty Drugs: Year in Review 2013

The year 2013 marked the approval of many new specialty medications, with drug debuts and new indications in areas including oncology, hepatitis C, HIV, multiple sclerosis, and inflammatory disease.

2013 proved to be another year in which specialty pharmaceuticals featured prominently in the FDA’s new drug approval and expanded indications list. This article provides a summary of specialty pharmacy—related FDA approvals and expanded indications for 2013. (Please consult product prescribing information for complete information.)

LEUKEMIAS AND LYMPHOMAS

Gleevec (imatinib mesylate; Novartis) received an expanded indication on January 25, 2013, to treat children newly diagnosed with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia in combination with chemotherapy. A kinase inhibitor initially approved in 2001, Gleevec is approved to treat many oncologic conditions.¹

A new indication was granted to Revlimid (lenalidomide; Celgene) on June 5, 2013, when the FDA approved its use in mantle cell lymphoma (MCL) in patients whose disease has relapsed or progressed after 2 prior therapies, 1 of which included bortezomib. It is given orally once daily for 21 days out of a 28-day cycle. Revlimid is only available through a restricted distribution program in conjunction with a risk evaluation and mitigation strategy (REMS) due to potential for embryo-fetal risk.²

Gazyva (obinutuzumab; Genentech) wasapproved on November 1, 2013, for use in combination with chlorambucil for the treatment of patients with previously untreated chronic lymphocytic leukemia. Gazyva is the first CD20-directed cytolytic antibody to be approved in the United States and is administered as an intravenous infusion.^3,4

On November 13, 2013, Imbruvica (ibrutinib; Pharmacyclics) was approved to treat patients with MCL who have received at least 1 prior therapy. Imbruvica is an inhibitor of Bruton’s tyrosine kinase and is dosed as 560 mg (four 140- mg capsules) taken orally once daily with a glass of water.^5,6

GASTROINTESTINAL TUMORS

Stivarga (rigorafenib; Bayer) is a oncedaily oral kinase inhibitor that was approved on February 25, 2013, for the additional indication of treating advanced gastrointestinal stromal tumors that cannot be surgically removed and that no longer respond to other FDA-approved treatments for the disease. Stivarga is given orally once daily for the first 21 days of each 28-day cycle and should be taken with food such as a low-fat breakfast. In 2012, Stivarga was approved for the treatment of metastatic colorectal cancer in patients who have previously been treated.^7,8

MELANOMA

On May 29, 2013, Tafinlar (dabrafenib; GlaxoSmithKline) received FDA approval for treatment of patients with unresectable or metastatic melanoma whose tumors express the BRAF V600E gene mutation. Tafinlar must be used with an FDAapproved genetic test that identifies the V600E or V600K mutation in the BRAF gene, such as the THxID BRAF test. The recommended dose is 150 mg orally twice daily taken at least 1 hour before or at least 2 hours after a meal. Tafinlar is not recommended for use in patients with wild-type BRAF melanoma and the product must be dispensed with an FDA-approved medication guide.⁹

Mekinist (trametinib; GlaxoSmith-Kline) was also approved by the FDA on May 29, 2013, to treat patients who have unresectable or metastatic melanoma whose tumors express the BRAF V600E or BRAF V600K gene mutations. It also requires use of an FDA-approved genetic test such as the THxID BRAF genetic test. Mekinist is the first mitogen-activated extracellular signal regulated kinase (MEK) inhibitor approved by the FDA and acts as a reversible inhibitor of MEK1 and MEK2 activation and of MEK1 and MEK2 kinase activity. The recommended dose is 2 mg orally once daily taken at least 1 hour before or at least 2 hours after a meal until disease progression or unacceptable toxicity. Mekinist is not indicated for the treatment of patients who have received a prior BRAF inhibitor therapy. It must be dispensed and stored in its original bottle with its manufacturer-supplied desiccant and should not be placed in pill boxes or stored anywhere other than in its original container.^9,10

LUNG CANCER

Tarceva (erlotinib; OSI Pharmaceuticals and Genentech) received an expanded indication on May 14, 2013, for initial (first-line) treatment of people with metastatic non-small cell lung cancer (NSCLC) whose tumors have certain epidermal growth factor receptor (EGFR)-activating mutations as detected by an FDA-approved test (such as the cobas EGFR Mutation Test). For NSCLC, it should be given as 150 mg orally on an empty stomach once daily. Tarceva was first approved in 2004 and is indicated for maintenance treatment of patients with locally advanced or metastatic NSCLC whose disease has not progressed after 4 cycles of platinum-based chemotherapy, for treatment of locally advanced or metastatic NSCLC after failure of at least 1 prior chemotherapy regimen, and for first-line treatment of patients with locally advanced, unresectable, or metastatic pancreatic cancer in combination with gemcitabine.^11,12

Gilotrif (afatinib; Boehringer Ingelheim) received FDA approval on July 12, 2013. It is indicated for the treatment of patients with metastatic NSCLC whose tumors have EGFR mutations. These mutations require detection by an FDA-approved test (such as the therascreen EGFR RGQ PCR kit) prior to treatment. Gilotrif has a recommended dose of 40 mg orally given once daily at least 1 hour before or 2 hours after a meal.¹³

PANCREATIC CANCER

Abraxane (nanoparticle albumin-bound [nab]-paclitaxel; Celgene) received an expanded indication on September 6, 2013, to treat patients with metastatic adenocarcinoma of the pancreas, as first-line treatment in combination with gemcitabine. For the pancreatic cancer indication, the recommended dosage of Abraxane is 125 mg/m2 infused intravenously over 30 to 40 minutes on days 1, 8, and 15 of each 28-day cycle. This approval joins previous Abraxane indications as a treatment option for breast cancer and non-small cell lung cancer.^14,15

THYROID CANCER

On November 22, 2013, the FDA approved Nexavar (sorafenib; Bayer) to treat locally recurrent or metastatic, progressive, differentiated thyroid carcinoma refractory to radioactive iodine treatment. The dose of Nexavar is 400 mg orally twice daily without food. It is a kinase inhibitor that decreases tumor cell proliferation. Nexavar was previously approved for the treatment of unresectable hepatocellular carcinoma and advanced renal cell carcinoma.^16,17

BREAST CANCER

Kadcyla (ado-trastuzumab emtansine; Genentech) is an intravenously administered HER2-targeted antibody and microtubule inhibitor conjugate indicated, as a single agent, for the treatment of patients with HER2-positive, metastatic breast cancer who previously received trastuzumab and a taxane separately or in combination. Patients should have either received prior therapy for metastatic disease or developed disease recurrence during or within 6 months of completing adjuvant therapy. Kadcyla is trastuzumab conjugated to a drug called DM1 that interferes with cancer cell growth. Kadcyla should be administered as an intravenous (IV) infusion over at least 30 minutes if well tolerated (infuse over 90 minutes for the first infusion) and given every 3 weeks until disease progression or unacceptable toxicity occurs.^18,19

On September 30, 2013, the FDA expanded the indication for Perjeta (pertuzumab; Genentech) to include its use as part of a complete treatment regimen for early breast cancer in combination with trastuzumab and docetaxel as neoadjuvant treatment of patients with HER2-positive, locally advanced, inflammatory, or earlystage breast cancer (either greater than 2 cm in diameter or node positive). Perjeta is generally given as a 60-minute IV infusion. It was previously approved for use in combination with trastuzumab and docetaxel for treatment of patients with HER2 positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease.^20,21

MULTIPLE MYELOMA

On February 8, 2013, the FDA approved Pomalyst (pomalidomide; Celgene), an oral capsule for the treatment of multiple myeloma in patients who have received at least 2 prior therapies (including bortezomib and lenalidomide) and have demonstrated disease progression within 60 days of completion of the last therapy. Pomalyst is administered once daily on days 1 to 21 of repeated 28-day cycles until disease progression. Pomalyst was approved with a REMS program. Prescribers must be certified with the Pomalyst REMS program by enrolling and complying with the REMS requirements. Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS program, must only dispense to patients who are authorized to receive the drug, and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS due to embryo-fetal risk.^22,23

BONE TUMORS

On June 13, 2013, the FDA approved an expanded indication for Xgeva (denosumab; Amgen) to treat adults and some adolescents with giant cell tumor of the bone (GCTB). GCTB is a rare and usually non-cancerous tumor that generally occurs in adults between the ages of 20 and 40 years. Xgeva, when used for GCTB, is administered as a subcutaneous injection in the upper arm, upper thigh, or abdomen at a dose of 120 mg every 4 weeks with additional 120-mg doses on day 8 and day 15 of the first month of therapy. Xgeva was previously approved in 2010 to prevent fractures when cancer has spread to the bones.^24,25

BLEEDING DISORDERS

The FDA approved Rixubis (coagulation factor IX [recombinant]; Baxter Healthcare Corporation) on June 26, 2013. Rixubis is an antihemophilic factor indicated for control and prevention of bleeding episodes, perioperative management, or routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients who are 16 years or older with hemophilia B. Rixubis marks the first recombinant coagulation factor IX indicated specifically for routine prophylaxis to prevent bleeding.²⁶

On October 16, 2013, the FDA approved Novoeight (turoctocog; Novo Nordisk) for use in adults and children with hemophilia A for the control and prevention of bleeding, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. Novoeight will be launched with the prefilled device called MixPro and it is expected to be launched in the United States in the second quarter of 2015.²⁷

Tretten (coagulation factor XIII A-Subunit [recombinant]; Novo Nordisk) was approved on December 23, 2013. It is the first recombinant product for use in the routine prevention of bleeding in adults and children who have congenital Factor XIII A-subunit deficiency. Tretten is a sterile freeze-dried powder to be reconstituted with diluent and injected intravenously

either by a health care professional or as a self-administered product.²⁸

TRANSPLANT

The FDA approved Astagraf XL (tacrolimus extended-release capsules; Astellas Pharma US, Inc) for oral use on July 19, 2013. It was approved for prophylaxis of organ rejection in patients receiving a kidney transplant with mycophenolate mofetil and corticosteroids, with or without basiliximab induction. Although various formulations of tacrolimus have been approved by the FDA since 1994, this is the first once-daily oral tacrolimus formulation for patients who have received a kidney transplant.²⁹

INFLAMMATORY DISEASES

Actemra (tocilizumab; Genentech) added an indication on April 30, 2013, when the FDA approved its use for the treatment of polyarticular juvenile idiopathic arthritis (PJIA) in patients 2 years and older with active disease. Actemra can be administered as stand-alone therapy or in conjunction with methotrexate. Actemra is a humanized interleukin-6 receptor antagonist and was previously approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to 1 or more disease-modifying antirheumatic drugs and for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients 2 years and older. For its RA, PJIA, and SJIA indications, Actemra is given as an IV infusion over 1 hour. Additionally, on October 21, 2013, the FDA approved a subcutaneous formulation of Actemra, which is available in a prefilled syringe, for the treatment of RA. The dosing for subcutaneous Actemra is as follows: for patients who weigh less than 100 kg the dose is 162 mg administered subcutaneously every other week followed by an increase to every week based on clinical response; for patients who weigh 100 kg or more the recommended dose is 162 mg administered subcutaneously every week.^30-32

Ilaris (canakinumab; Novartis) also added an indication on May 10, 2013, when the FDA approved its use for the treatment of active SJIA in patients 2 years and older. Ilaris is an interleukin-1 beta inhibitor given as a once-monthly subcutaneous injection. It was previously approved in the United States for the treatment of cryopyrin-associated periodicsyndromes.³³

In addition, Simponi (golimumab; Janssen) was granted an additional indication on May 15, 2013, when the FDA approved its use to treat patients with moderate to severe ulcerative colitis that is resistant to prior treatments or requires continuous steroid therapy. Simponi was previously available in the United States to treat RA, psoriatic arthritis, and ankylosing spondylitis. Simponi dosing for ulcerative colitis differs from that of the approved dosing for the other indications. The recommended dose to treat moderate to severe ulcerative colitis is 200 mg as a subcutaneous injection initially followed by 100 mg at week 2 and then 100 mg every 4 weeks.^34,35

Simponi Aria (golimumab; Janssen) was approved by the FDA on July 18, 2013, as a new infused dosage form of golimumab for the treatment of adults with moderately to severely active RA in combination with methotrexate. The approval of Simponi Aria marked the availability of the first anti—tumor necrosis factor (TNF) infusion therapy in more than a decade. The recommended dosage for RA is 2 mg/ kg given as an intravenous infusion over 30 minutes at weeks 0 and 4, and then every 8 weeks thereafter.³⁶

On September 20, 2013, Stelara (ustekinumab; Janssen) was granted an additional indication when the FDA approved its use either alone or in combination with methotrexate to treat patients with active psoriatic arthritis. For the treatment of psoriatic arthritis, Stelara is administered as a 45-mg subcutaneous injection at week 0, week 4, and every 12 weeks thereafter. It is a human interleukin-12 and interleukin-23 antagonist and was previously approved for the treatment of adult patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy.³⁷

Cimzia (certolizumab pegol; UCB) was granted 2 additional indications by the FDA in 2013. On September 30, it was approved for treatment of adult patients with active psoriatic arthritis and on October 17 was granted approval for the treatment of adults with active ankylosing spondylitis. The dose of Cimzia for psoriatic arthritis is 400 mg given subcutaneously initially and at week 2 and week 4, followed by 200 mg every other week; for maintenance dosing, 400 mg every 4 weeks can be considered. The dose of Cimzia for ankylosing spondylitis is 400 mg initially and at weeks 2 and 4, followed by 200 mg every other week or 400 mg every 4 weeks. Cimzia was previously approved in the United States for the treatment of adult patients with moderately to severely active RA and to lessen the signs and symptoms of moderately to severely active Crohn’s disease in adult patients who have not responded to other treatments.^38-40

On October 14, 2013, the FDA approved Otrexup (methotrexate self-administered injectable; Antares Pharma). This product is the first subcutaneous methotrexate (MTX) FDA-approved for once weekly self-administration with an auto injector. Otrexup is indicated for adults with severe active RA who have had an insufficient therapeutic response to or are intolerant of an adequate trial of first-line therapy including full dose non-steroidal anti-inflammatory agents, children with active PJIA, or symptomatic control of severe recalcitrant, disabling psoriasis that is not adequately responsive to other forms of therapy. Otrexup is administered once weekly as a subcutaneous injection in the abdomen or thigh.⁴¹

CYSTIC FIBROSIS

Tobi Podhaler (tobramycin; Novartis) is a new delivery system for tobramycin for inhalation approved on March 22, 2013. It is indicated for the management of cystic fibrosis patients with Pseudomonas aeruginosa. The Tobi Podhaler is a handheld plastic inhaler device that contains capsules of a dry powder formulation of tobramycin. The powder from the capsules is inhaled twice daily using the Podhaler device. Therapy is typically given on alternating cycles of 28 days on therapy and 28 days off therapy.⁴²

HIV

Sustiva (efavirenz; Bristol-Myers Squibb) was granted an expanded patient population on May 3, 2013, when the FDA approved a supplemental new drug application that included dosing recommendations for HIV-1 infected pediatric patients aged 3 months to 3 years and weighing at least 3.5 kilograms. As part of the approval for this pediatric population, a oncedaily option is available that includes capsule sprinkle administration for those who cannot swallow capsules or tablets. Sustiva was previously approved in the United States in 1998 to treat HIV-1-infected adults and children 3 years or older and weighing at least 10 kg.⁴³

On August 12, 2013, the FDA approved Tivicay (dolutegravir; GlaxoSmithKline), a new antiretroviral agent to treat HIV-1 infection. Tivicay is an integrase strand transfer inhibitor (INSTI) which interferes with an enzyme necessary for HIV replication and must be taken daily in combination with other antiretroviral drugs. The FDA approved Tivicay for treatment-naïve HIV-infected adults and treatment-experienced HIV-infected adults including those previously treated with other INSTIs, as well as approval for a pediatric indication which supports its use in treatment-naïve or treatment-experienced children 12 years and older weighing at least 40 kg who have not had a previous exposure to INSTIs. Dosing is dependent on patient specific parameters and begins at 50 mg once daily with or without food.^44,45

HEPATITIS C

Olysio (simeprevir; Janssen) was approved as a new option to treat hepatitis C virus (HCV) on November 22, 2013. Olysio is a hepatitis C virus NS3/4A protease inhibitor indicated for the treatment of chronic hepatitis C infection as a component of a combination antiviral treatment regimen. Olysio is not to be used as monotherapy and its efficacy has been established in combination with peginterferon alfa and ribavirin in HCV genotype 1 infected patients. 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. Olysio is dosed as one 150-mg capsule taken once daily with food and should be administered with both peginterferon alfa and ribavirin. The recommended treatment duration of Olysio with peginterferon alfa and ribavirin is 12 weeks, followed by either 12 or 36 additional weeks of peginterferon alfa and ribavirin depending on prior response status.^46,47

On December 6, 2013, the FDA approved Sovaldi (sofosbuvir; Gilead) as another new option to treat HCV. Sovaldi is an HCV nucleotide analogue NS5B polymerase inhibitor indicated for the treatment of chronic hepatitis C infection as a component of a combination antiviral treatment regimen. Sovaldi has demonstrated efficacy in patients with HCV genotype 1, 2, 3, or 4 infection and is given as one 400-mg tablet once daily with or without food. Sovaldi should be used in combination with ribavirin or in combination with pegylated interferon plus ribavirin. The recommended combination and duration of therapy is dependent on the genotype of the virus and the prescribing information should be consulted for specific details.^48,49

MULTIPLE SCLEROSIS

Tecfidera (dimethyl fumarate; Biogen Idec) was approved by the FDA on March 27, 2013, to treat adults with relapsing forms of multiple sclerosis. The recommended starting dose of Tecfidera is 120 mg orally twice a day for 7 days. After 7 days, a maintenance dose of 240 mg twice a day is recommended.⁵⁰

HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

The approval of Kynamro (mipomersen sodium; Genzyme Corp) injection on January 29, 2013, meant another option in the treatment of homozygous familial hypercholesterolemia (HoFH). HoFH is an inherited condition that affects approximately 1 in 1 million people in the United States. Kynamro helps to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and nonhigh density lipoprotein-cholesterol (non HDL-C). The FDA approved Kynamro with a REMS program including elements to assure safe use, such as prescriber and pharmacy certification, and documentation of safe-use conditions, which requires a prescription authorization form for each new prescription.⁵¹

UREA CYCLE DISORDERS

On February 1, 2013, the FDA approved Ravicti (glycerol phenylbutyrate; Hyperion Therapeutics) for the chronic management of some urea cycle disorders in patients 2 years and older. Ravicti helps dispose of ammonia in the body. It is a liquid taken 3 times daily with meals and is intended for patients whose urea cycle disorder cannot be managed by a proteinrestricted diet or amino acid supplements alone.⁵²

IRON OVERLOAD

On January 23, 2013, the FDA approved the expanded use of Exjade (deferasirox; Novartis Oncology) to treat patients 10 years and older who have chronic iron overload due to the genetic blood disorder non-transufsion-dependent thalassemia. Exjade was previously approved for treatment of chronic iron overload due to blood transfusions in patients 2 years and older. At that time, the FDA also authorized marketing of FerriScan (Resonance Health) as an imaging companion diagnostic for Exjade. FerriScan measures liver iron concentration (LIC) non-invasively using MRI technology.⁵³

PULMONARY ARTERIAL HYPERTENSION

On October 9, 2013, the FDA approved Adempas (riociguat; Bayer) for adults with 2 different forms of pulmonary hypertension. Adempas is a stimulator of soluble guanylate cyclase that helps arteries relax to increase blood flow and decrease blood pressure. It is indicated for adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class, as well as for adults with pulmonary arterial hypertension (PAH) (WHO Group 1) to improve exercise capacity, improve WHO functional class, and delay clinical worsening. Adempas tablets are typically given 3 times a day and were approved with a REMS program due to their risk of fetal toxicity.⁵⁴

Opsumit (macitentan; Actelion) was approved on October 18, 2013, as a new endothelin receptor antagonist to treat adults with PAH WHO Group I in order to delay disease progression. The recommended dose of Opsumit is 10 mg orally once daily. Opsumit was approved with a REMS program due to its risk of fetal toxicity.^55,56

On December 20, 2013, the FDA approved Orenitram (treprostinil) Extended- Release Tablets for the treatment of PAH in WHO Group I patients to improve exercise capacity. This marks the first time that an orally administered prostacyclin analogue has been approved. Orenitram contains the same active ingredient as Remodulin (treprostinil) Injection and Tyvaso (treprostinil) Inhalation Solution. Orenitram is dosed twice a day with food, but the total daily dose can be divided and given 3 times daily with food. The dose should be increased as tolerated to achieve optimal clinical response, and the maximum dose is determined by tolerability.⁵⁷

NEPHROPATHIC CYSTINOSIS

On April 30, 2013, the FDA approved Procysbi (cysteamine bitartrate; Raptor Pharmaceuticals) for the management of nephropathic cystinosis in children and adults. Cystinosis is fatal if not treated early in childhood and causes cystine buildup in every cell of the body. This cystine buildup causes kidney problems, which can cause the body to lose sugar, protein, and salts through the urine. It is a rare genetic condition which affects approximately 500 people in the United States. Procysbi is a delayed-release capsule to be taken every 12 hours.⁵⁸ SPT

References

• FDA approves Gleevec for children with acute lymphoblastic leukemia [press release]. FDA website. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm336868.htm. Published January 25, 2013. Accessed January 7, 2014.
• US Food and Drug Administration approves Revlimid (lenalidomide) for the treatment of patients with relapsed or refractory mantle cell lymphoma. Celgene; June 5, 2013. Accessed January 7, 2014.
• FDA approves Gazyva for chronic lymphocytic leukemia [press release]. FDA website; November 1, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm373209.htm. Accessed January 7, 2014.
• Gazyva [prescribing information]. Genentech; November 2013. www.gene.com/download/pdf/gazyva_prescribing.pdf. Accessed January 7, 2014.
• Imbruvica [prescribing information]. Pharmacyclics; November 2013. www.imbruvica.com/downloads/Prescribing_Information.pdf. Accessed January 7, 2014.
• FDA approves Imbruvica for rare blood cancer [press release]. FDA website; November 13, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm374761.htm. Accessed January 7, 2014.
• FDA approves Stivarga for advanced gastrointestinal stromal tumors [press release]. FDA website; February 25, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm340958.htm. Accessed January 7, 2014.
• Stivarga [prescribing information]. Bayer; August 2013. http://labeling.bayerhealthcare.com/html/products/pi/Stivarga_PI.pdf. Accessed January 7, 2014.
• Two new GSK oral oncology treatments, BRAF-inhibitor Tafinlar (dabrafenib) capsules and the first MEK-inhibitor Mekinist (trametinib) tablets, approved by FDA as single-agent therapies [press release]. GlaxoSmithKline website. http://us.gsk.com/html/media-news/pressreleases/2013/2013-pressrelease-1350998.htm. Published May 29, 2013. Accessed January 7, 2014.
• Mekinist [prescribing information]. GlaxoSmithKline; May 2013. http://us.gsk.com/products/assets/us_mekinist.pdf. Accessed January 7, 2014.
• FDA approves Tarceva (erlotinib) tablets and cobas EGFR mutation test for specific type of lung cancer [press release]. Genentech; May 14, 2013. www.gene.com/media/press-releases/14407/2013-05-14/fda-approves-tarceva-erlotinib-tablets-a. Accessed January 7, 2014.
• Tarceva [prescribing information]. Genentech; October 2013. www.gene.com/download/pdf/tarceva_prescribing.pdf. Accessed January 7, 2014.
• FDA approves new treatment for a type of late-stage lung cancer [press release]. FDA website; July 14, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm360499.htm. Accessed January 7, 2014.
• FDA approves Abraxane for late-stage pancreatic cancer [press release]. FDA website; September 6, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm367442.htm. Accessed January 7, 2014.
• Abraxane [prescribing information]. Celgene; October 2013. www.abraxane.com/downloads/Abraxane_PrescribingInformation.pdf. Accessed January 7, 2014.
• FDA approves Nexavar to treat type of thyroid cancer [press release]. FDA website; November 22, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm376443.htm. Accessed January 7, 2014.
• Nexavar [prescribing information]. Bayer; November 2013. http://labeling.bayerhealthcare.com/html/products/pi/Nexavar_PI.pdf. Accessed January 7, 2014.
• FDA approves new treatment for late-stage breast cancer [press release]. FDA website; February 22, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm340704.htm. Accessed January 7, 2014.
• Kadcyla [prescribing information]. Genentech; May 2013. www.gene.com/download/pdf/kadcyla_prescribing.pdf. Accessed January 7, 2014.
• FDA approves Perjeta for neoadjuvant breast cancer treatment [press release]. FDA website; September 30, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm370393.htm. Accessed January 7, 2014.
• Perjeta [prescribing information]. Genentech; September 2013. www.gene.com/download/pdf/perjeta_prescribing.pdf. Accessed January 7, 2014.
• FDA approves Pomalyst for advanced multiple myeloma [press release]. FDA website; February 8, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm338895.htm. Accessed January 7, 2014.
• Pomalyst [prescribing information]. Celgene; February 2013. www.pomalyst.com/wp-content/uploads/2013/08/prescribing_information.pdf. Accessed January 7, 2014.
• FDA approves Xgeva to treat giant cell tumor of the bone [press release]. FDA website; June 13, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm356528.htm. Accessed January 7, 2014.
• Xgeva [prescribing information]. Amgen; August 2013. http://pi.amgen.com/united_states/xgeva/xgeva_pi.pdf. Accessed January 7, 2014.
• FDA approves first recombinant coagulation factor IX that is specifically indicated for routine use in preventing bleeding episodes (prophylaxis) [press release]. FDA website; June 27, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm358918.htm. Accessed January 7, 2014.
• Novoeight receives approval from the FDA [press release]. Hemophilia Federation of America; October 16, 2013. www.hemophiliafed.org/news-stories/2013/10/novoeight-receives-approval-fda. Accessed January 7, 2014.
• FDA approves Tretten to treat rare genetic clotting disorder [press release]. FDA website; December 23, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm379696.htm. Accessed January 7, 2014.
• Astellas announces FDA approval of Astagraf XL (tacrolimus extended-release capsules) for the prophylaxis of organ rejection in adult kidney transplant recipients [press release]. Astellas; July 19, 2013. http://newsroom.astellas.us/2013-07-19-Astellas-Announces-FDA-Approval-of-ASTAGRAF-XL-tacrolimus-extended-release-capsules-for-the-Prophylaxis-of-Organ-Rejection-in-Adult-Kidney-Transplant-Recipients. Accessed January 7, 2014.
• FDA approves Actemra for children living with a rare form of arthritis [press release]. Genentech; April 30, 2013. www.gene.com/media/press-releases/14387/2013-04-30/fda-approves-actemra-for-children-living. Accessed January 7, 2014.
• Genentech gains FDA approval for new subcutaneous formulation of Actemra for use in adult patients living with moderately to severely active rheumatoid arthritis [press release]. Genentech; October 21, 2013. www.gene.com/media/press-releases/14557/2013-10-21/genentech-gains-fda-approval-for-new-sub. Accessed January 7, 2014.
• Actemra [prescribing information]. Genentech; October, 2013. www.gene.com/gene/products/information/actemra/pdf/pi.pdf. Accessed January 7, 2014.
• Novartis drug Ilaris approved by FDA to treat active systemic juvenile idiopathic arthritis, a serious form of childhood arthritis [press release]. Novartis; May 10, 2013. www.novartis.com/newsroom/media-releases/en/2013/1700796.shtml. Accessed January 7, 2014.
• FDA approves Simponi to treat ulcerative colitis [press release]. FDA website; May 15, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm352383.htm. Accessed January 7, 2014.
• Simponi [prescribing information]. Janssen; November 2013. www.simponi.com/shared/product/simponi/prescribing-information.pdf. Accessed January 7, 2014.
• Simponi Aria (golimumab) for infusion receives FDA approval for treatment of moderately to severely active rheumatoid arthritis [press release]. Janssen Biotech; July 18, 2013. www.prnewswire.com/news-releases/simponi-aria-golimumab-for-infusion-receives-fda-approval-for-treatment-of-moderately-to-severely-active-rheumatoid-arthritis-216075111.html. Accessed January 8, 2014.
• Stelara (Ustekinumab) receives FDA approval to treat active psoriatic arthritis [press release]. Janssen Biotech; September 23, 2013. www.investor.jnj.com/releasedetail.cfm?ReleaseID=792461. Accessed January 8, 2014.
• Cimzia (certolizumab pegol) approved by the US FDA for treatment of adult patients with active psoriatic arthritis [press release]. UCB; September 30, 2013. www.prnewswire.com/news-releases/cimzia-certolizumab-pegol-approved-by-the-us-fda-for-treatment-of-adult-patients-with-active-psoriatic-arthritis-225778051.html. Accessed January 8, 2014.
• Cimzia (certolizumab pegol) approved by FDA for treatment of adults with active ankylosing spondylitis [press release]. UCB; October 18, 2013. www.prnewswire.com/news-releases/cimzia-certolizumab-pegol-approved-by-fda-for-treatment-of-adults-with-active-ankylosing-spondylitis-228312901.html. Accessed January 8, 2014.
• Cimzia [prescribing information]. UCB; October 2013. www.cimzia.com/pdf/Prescribing_Information.pdf. Accessed January 8, 2014.
• Otrexup (methotrexate) injection approved by FDA a new treatment for adults with rheumatoid arthritis, children with polyarticular idiopathic arthritis, and adults with psoriasis [press release]. Antares Pharma; October 14, 2013. www.businesswire.com/news/home/20131014005610/en/OTREXUP%E2%84%A2-methotrexate-Injection-Approved-FDA-Treatment-Adults. Accessed January 8, 2014.
• FDA approves TOBI Podhaler to treat a type of bacterial lung infection in cystic fibrosis patients [press release]. FDA website; March 22, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm345123.htm. Accessed January 8, 2014.
• Bristol-Myers Squibb receives US FDA sNDA approval for use of Sustiva (efavirenz) in HIV-1 infected pediatric patients [press release]. Bristol Myers Squibb; May 3, 2013. http://news.bms.com/press-release/rd-news/bristol-myers-squibb-receives-us-fda-snda-approval-use-sustiva-efavirenz-hiv-1. Accessed January 8, 2014.
• Tivicay [prescribing information]. Viiv; August 2013. www.viivhealthcare.com/media/58599/us_tivicay.pdf. Accessed January 8, 2014.
• 45. FDA approves new drug to treat HIV infection [press release]. FDA website; August 12, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm364744.htm. Accessed January 8, 2014.
• FDA approves new treatment for hepatitis C virus [press release]. FDA website; November 22, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm376449.htm. Accessed January 8, 2014.
• Olysio [prescribing information]. Janssen Therapeutics; November 2013. www.olysio.com/shared/product/olysio/prescribing-information.pdf. Accessed January 8, 2014.
• FDA approves Sovaldi for chronic hepatitis C [press release]. FDA website; December 6, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm377888.htm. Accessed January 8, 2014.
• Sovaldi [prescribing information]. Gilead; December 2013. www.gilead.com/~/media/Files/pdfs/medicines/liver-disease/sovaldi/sovaldi_pi.pdf. Accessed January 8, 2014.
• Tecfidera [prescribing information]. Biogen Idec, May 2013. www.tecfidera.com/pdfs/full-prescribing-information.pdf. Accessed January 8, 2014.
• FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder [press release]. FDA website; January 29, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337195.htm. Accessed January 8, 2014.
• FDA approves new drug for the chronic management of some urea cycle disorders [press release]. FDA website; February 1, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337639.htm. Accessed January 8, 2014.
• FDA approves Exjade to remove excess iron in patients with genetic blood disorder [press release]. FDA website; January 23, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm336478.htm. Accessed January 8, 2014.
• FDA approves Adempas to treat pulmonary hypertension [press release]. FDA website; October 8, 2013. www.fda.gov/newsevents/newsroom/pressannouncements/ucm370866.htm. Accessed January 8, 2014.
• FDA approves Opsumit to treat pulmonary arterial hypertension [press release]. FDA website; October 18, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm371362.htm. Accessed January 8, 2014.
• Opsumit [prescribing information]. Actelion; October 2013. www.opsumitrems.com/pdf/OPSUMIT-Full-Prescribing-Information.pdf. Accessed January 8, 2014.
• FDA approves Orenitram (treprostinil) extended-release tablets for the treatment of pulmonary arterial hypertension [press release]. United Therapeutics; December 20, 2013. http://ir.unither.com/releasedetail.cfm?ReleaseID=815500. Accessed January 8, 2014.
• FDA approves Procysbi for rare genetic condition [press release]. FDA website; April 30, 2013. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm350091.htm. Accessed January 8, 2014.

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 author alone and not of Managed Health Care Associates, Inc.

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, and 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.