Near-Term Specialty Pipeline Highlights

A significant number of new specialty medications are on track to be approved in 2013, and some will provide increased competition in certain therapy classes.

The FDA is on track to approve a significant number of specialty medications in 2013, as they did in 2012. Many of the new specialty drugs expected to be launched this year are novel; however, several will be similar to other medications already on the market. The increased competition in certain specialty therapy classes will be welcomed by patients, prescribers, and payers alike. Patients and prescribers will have more therapies available to treat chronic and oftentimes debilitating conditions. In addition, more options will allow payers to designate preferred medications in therapy classes to help mitigate rising specialty drug costs.

Kynamro (mipomersen sodium; Isis/Genzyme) was the first specialty drug approved this year. It is administered as a monthly subcutaneous (SQ) injection to decrease low-density lipoprotein (LDL) cholesterol in patients with the rare condition called homozygous familial hypercholesterolemia. It competes with Juxtapid (lomitapide; Aegerion), a daily oral capsule approved in December 2012 for the same condition. Genentech’s Kadcycla (ado-trastuzumab emtansine) was approved in February; it is an infused antibody-drug conjugate that combines Herceptin with a chemotherapeutic agent to treat HER2-positive metastatic breast cancer. The highly anticipated Tecfidera (dimethyl fumarate; Biogen Idec) gained FDA approval in March to treat patients with multiple sclerosis. It is the third oral competitor in this market following Gilenya (fingolimod; Novartis) and Aubagio (teriflunomide; Sanofi).

The FDA will be busy for the remainder of the year as it continues its review of several specialty medications including those to treat cancer, hepatitis C, pulmonary arterial hypertension, bleeding disorders, and many other orphan conditions. More information about selected specialty pipeline medications can be found below.

Cancer

Tovok (afatinib) is Boehringer Ingelheim’s oral, targeted therapy that inhibits epidermal growth factor receptor (EGFR) and human epidermal receptor 2 (HER2) for the first-line treatment of EGFR-mutation positive advanced non—small cell lung cancer. Approximately 10% of lung cancer patients in the United States have EGFR mutations. In a clinical trial, Tovok improved progression-free survival by nearly 1 year compared with 7 months in patients taking standard chemotherapy. Tovok will primarily compete with Tarceva (erlotinib; Genentech/Astellas), a similar medication that has been on the market since 2004. Approval of Tovok is expected by mid-July. Pharmacogenetic tests will be available to identify appropriate patients for treatment with Tovok (and Tarceva).

Xofigo (radium Ra 223 dichloride) is Algeta and Bayer’s radiopharmaceutical that delivers alpha radiation specifically to bone metastases without affecting normal bone marrow. It was approved May 15, 2013, to treat bone metastases in patients with castration-resistant prostate cancer (CRPC). In a pivotal trial, Xofigo improved overall survival by 3.5 months compared with best supportive care (14.9 months vs 11.3 months). Xofigo is administered as a monthly IV infusion. It will primarily compete with Xgeva (denosumab; Amgen), but will also compete with other drugs that improve overall survival in patients with CRPC, including Jevtana (cabazitaxel; Sanofi), Provenge (sipuleucel-T; Dendreon), Xtandi (enzalutamide; Astellas/Medivation) and Zytiga (abiraterone acetate; Ortho Biotech). Approval of Xofigo occurred 3 months ahead of schedule.

Hepatitis C

Simeprevir is a hepatitis C virus (HCV) protease inhibitor that is pending approval for treatment-naïve and treatment-experienced patients with genotype 1 chronic hepatitis C with compensated liver disease. Approximately 3.2 million Americans have hepatitis C, a chronic liver infection that can cause cirrhosis and liver cancer if left untreated. Janssen and Medivir’s simeprevir is taken as one 150-mg capsule once daily for 12 weeks. It is given in addition to therapy with pegylated interferon (SQ) and ribavirin (oral), which are taken for 24 to 48 weeks. Simeprevir will compete with Incivek (telaprevir; Vertex) and Victrelis (boceprevir; Merck), which are oral HCV protease inhibitors that have been on the market since May 2011. The FDA is expected to approve simeprevir by late November.

Sofosbuvir is Gilead’s HCV polymerase inhibitor and is expected to be approved by early December for treatment-naïve and treatment-experienced patients with genotypes 1-6 chronic hepatitis C. For genotypes 1, 4, 5, and 6 chronic hepatitis C, sofosbuvir is taken as one 400-mg tablet once daily with pegylated interferon and ribavirin for 12 weeks. For genotypes 2 and 3 chronic hepatitis C, sofosbuvir is taken with ribavirin for 12 weeks. This will be the first all-oral (interferon-free) regimen approved for hepatitis C. By the end of next year, a single tablet formulation of sofosbuvir with ledipasvir (NS5a inhibitor) is expected to reach the market for genotype 1 chronic hepatitis C. This all-oral, once daily regimen will be the first interferon-free regimen for the most common and difficult-to-treat hepatitis C population. There are several more oral, direct antiviral agents in the pipeline. It is a very dynamic and quickly evolving market.

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About the Author

Aimee Tharaldson, PharmD, is a senior clinical consultant in the emerging therapeutics department at Express Scripts. She is responsible for monitoring and analyzing the specialty pharmaceutical pipeline. The emerging therapeutics department produces several proprietary reports on the pipeline for use by Express Scripts’ employees and clients. It is also responsible for the safety program that alerts patients, physicians, and clients to important information regarding serious drug safety alerts and market withdrawals. She contributes to Express Scripts’ Drug Trend Report and plays a key role in developing and maintaining Express Scripts’ specialty drug list. She received her doctor of pharmacy degree from the University of Minnesota, College of Pharmacy, and completed a pharmacy practice residency at the Minneapolis VA Medical Center.