Specialty Pipeline Highlights

THE FDA APPROVED 33 SPECIALTY DRUGS IN 2015, which is an increase in the number of specialty approvals compared with 2014 and 2013 (27 and 19, respectively). Many of these medications targeted orphan conditions and cancer.

Additionally, several new breakthrough therapies were approved last year. Among the most significant specialty medications given the green light in 2015 were a pair of proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors that lower cholesterol.

Two-thirds of the specialty drugs approved last year were for the treatment of rare conditions. Three new orphan breakthrough therapies approved in 2015 were Orkambi (lumacaftor/ivacaftor; Vertex), Strensiq (asfotase alfa; Alexion), and Kanuma (sebelipase alfa; Alexion).

Orkambi is an oral combination tablet that treats the underlying disease in cystic fibrosis patients aged 12 and older who have 2 copies of the F508del mutation in their cystic fibrosis transmembrane conductance regulator gene. Approximately 8500 patients in the United States are candidates for treatment with Orkambi.

Strensiq and Kanuma are injected enzyme replacement therapies that treat hypophosphatasia and lysosomal acid lipase deficiency, respectively. Both genetic diseases affect less than 20 patients per 1 million in the general population. All 3 breakthrough orphan therapies cost approximately $300,000 per year.

Among the drugs approved by the FDA, last year, were 15 new cancer therapies. Five targeted breakthrough cancer medications approved in 2015 were Ibrance (palbociclib; Pfizer), Tagrisso (osimertinib; AstraZeneca), Darzalex (daratumumab; Janssen Biotech), Empliciti (elotuzumab; Bristol-Myers Squibb), and Alecensa (alectinib; Genentech).

Ibrance is an oral cyclin-dependent kinase 4 and 6 inhibitor used in combination with Femara (letrozole; Novartis) for the first-line treatment of postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2 negative (ER+/HER2-) advanced breast cancer.

Tagrisso, an oral epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), is the first medication approved to treat patients with non-small cell lung cancer (NSCLC) who develop resistance to first-line TKIs due to the development of the EGFR T790M mutation.

Darzalex and Empliciti are infused biologic drugs that are now available to treat patients with relapsed or refractory multiple myeloma. Alecensa is an oral TKI approved for the second-line treatment of patients with anaplastic lymphoma kinase (ALK) positive NSCLC.

Most of the new, targeted cancer therapies cost between $100,000 and $150,000 per year. Sanofi and Regeneron’s Praluent (alirocumab) and Amgen’s Repatha (evolocumab) are PCSK9 inhibitors approved last summer for the treatment of high cholesterol among patients with heterozygous familial hypercholesterolemia or patients with clinical atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein (bad) cholesterol.

Repatha was also approved to treat patients with homozygous familial hypercholesterolemia. Both drugs, which are administered by subcutaneous injection, are used along with diet and maximally tolerated HMG co-reductase inhibitor (statin) therapy. Both treatments cost approximately $14,000 per year.

In 2016, watch for the approvals of new drugs to treat Duchenne muscular dystrophy (DMD), as well as breakthrough therapies for cancer and more options to treat hepatitis C. More information about selected specialty pipeline medications can be found below.

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