In 2013, many new medications and new indications were approved.
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. This article provides a selection of specialty pharmacy– related FDA approvals and expanded indications for 2013.


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) was approved 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


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 once-daily 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.7

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 for a pediatric indication 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.8,9


Olysio (simeprevir; Janssen) was approved as a new option to treat hepatitis C virus (HCV) on November 22, 2013. Olysio is an HCV 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.10,11

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 HCV 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.12,13

Stacey Ness, PharmD, RPh, CSP, MSCS, AAHIVP, is a multiple sclerosis–certified specialist, credentialed HIV pharmacist, and certified specialty pharmacist, and is the director of specialty clinical services at Managed Health Care Associates, Inc, a health care services organization in Florham Park, New Jersey. Dr. Ness is an associate editor for Specialty Pharmacy Times.

  1. FDA approves Gleevec for children with acute lymphoblastic leukemia [press release]. FDA website. Published January 25, 2013. Accessed January 7, 2014.
  2. 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.
  3. FDA approves Gazyva for chronic lymphocytic leukemia [press release]. FDA website; November 1, 2013. Accessed January 7, 2014.
  4. Gazyva [prescribing information]. Genentech; November 2013. Accessed January 7, 2014.
  5. Imbruvica [prescribing information]. Pharmacyclics; November 2013. Accessed January 7, 2014.
  6. FDA approves Imbruvica for rare blood cancer [press release]. FDA website; November 13, 2013. Accessed January 7, 2014.
  7. 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. Accessed January 8, 2014.
  8. Tivicay [prescribing information]. Viiv; August 2013. Accessed January 8, 2014.
  9. FDA approves new drug to treat HIV infection [press release]. FDA website; August 12, 2013. Accessed January 8, 2014.
  10. FDA approves new treatment for hepatitis C virus [press release]. FDA website; November 22, 2013. Accessed January 8, 2014.
  11. Olysio [prescribing information]. Janssen Therapeutics; November 2013. Accessed January 8, 2014.
  12. FDA approves Sovaldi for chronic hepatitis C [press release]. FDA website; December 6, 2013. Accessed January 8, 2014.
  13. Sovaldi [prescribing information]. Gilead; December 2013. Accessed January 8, 2014.