Specialty Pipeline Highlights

The FDA has approved several new specialty medications thus far in 2017. Sanofi and Regeneron’s dupilumab (Dupixent) is a new biologic drug that was approved in March to treat patients with moderate to severe atopic dermatitis whose disease cannot be adequately controlled with topical prescription therapies. It is administered by subcutaneous (SC) injection every 2 weeks and costs approximately $37,000 per year.

Genentech’s ocrelizumab (Ocrevus), also approved in March, is the first drug indicated to treat patients with primary progressive multiple sclerosis (PPMS). It was also approved to treat patients with relapsing forms of multiple sclerosis (MS). Ocrevus is administered via intravenous (IV) infusion every 6 months and costs approximately $65,000 per year. PPMS affects up to 15% of the 400,000 patients in the United States with MS. Patients with PPMS typically have worsening disease without remissions, and it generally causes more disability than other forms of MS.

In April, the FDA approved 2 new oral drugs known as vesicular monoamine transporter 2 (VMAT2) inhibitors. Teva’s deutetrabenazine (Austedo) was approved to treat chorea associated with Huntington’s disease (HD), a fatal neurodegenerative disorder.

There are approximately 35,000 patients in the United States with HD, approximately 90% of whom have HD-associated chorea, which is characterized by abnormal, involuntary movements. Tetrabenazine (Xenazine) is another VMAT2 inhibitor and has been available since 2008 for treatment of HD. It is dosed 3-times daily compared with twice daily for Austedo.

Neurocrine Biosciences’ once-daily valbenazine (Ingrezza) was the second VMAT2 inhibitor approved in April. It is indicated to treat patients with tardive dyskinesia (TD), which is characterized by abnormal, involuntary movements in patients exposed to certain drugs, particularly those that block dopamine in the brain. As many as 500,000 patients in the United States have TD. Austedo is expected to gain an expanded indication for TD by August 30, 2017. Austedo and Ingrezza cost approximately $60,000 per year.

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Several breakthrough cancer drugs were approved in the first half of 2017. New infused programmed cell death receptor ligand-1 inhibitors include EMD Serono and Pfizer’s avelumab (Bavencio) and AstraZeneca’s durvalumab (Imfinzi). Bavencio was approved in March to treat metastatic Merkel cell carcinoma, a rare and aggressive skin cancer that is diagnosed in approximately 1600 Americans annually. Imfinzi was approved in May to treat patients with metastatic urothelial carcinoma, the most common type of bladder cancer. Approximately 79,000 patients are diagnosed with bladder cancer each year in the United States.

New oral targeted cancer therapies include: ribociclib (Kisqali) as a first-line treatment for postmenopausal women who have metastatic or advanced hormone receptor positive, human epidermal growth factor receptor-2 negative breast cancer; niraparib (Zejula) for maintenance treatment of women who have had a partial response to platinum-based chemotherapy for recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer; midostaurin (Rydapt) for the first-line treatment of adults with FMS-like tyrosine kinase 3 mutation—positive (FLT3-positive) acute myeloid leukemia; and brigatinib (Alunbrig) for treating patients who have metastatic anaplastic lymphoma kinase–positive non–small cell lung cancer and who have progressed on, or are intolerant to, crizotinib (Xalkori).

Most new cancer medications cost more than $150,000 per year. Over the next several months, watch for more competitors in the inflammatory conditions market. Additionally, new pan-genotypic regimens for hepatitis C and the first chimeric antigen receptor T (CAR T) cell therapies for cancer are expected to gain approval.

Inflammatory Conditions

New competitors are expected in the rheumatoid arthritis (RA) and psoriasis markets. Sanofi and Regeneron’s sarilumab (Kevzara) is an interleukin-6 (IL-6) receptor inhibitor recently approved for the treatment of RA. It is administered as a SC injection every other week. The results of a clinical study show that Kevzara significantly improved the signs and symptoms of RA compared with adalimumab (Humira).

Kevzara will compete with Genentech’s tocilizumab (Actemra) an IV and SC IL-6 inhibitor, which was approved on May 22. Janssen and GlaxoSmithKline’s sirukumab is another IL-6 inhibitor that is expected to be approved by September 23, 2017. Janssen’s guselkumab is an interleukin-23 (IL-23) inhibitor that is expected to be approved by July 14, 2017, for the treatment of patients with moderate to severe plaque psoriasis. After induction dosing, it is administered every 8 weeks by SC injection. Guselkumab demonstrated improved skin clearance compared with Humira in 2 late-phase studies.

Sun Pharmaceuticals’ tildrakizumab is another IL-23 inhibitor that is in development for psoriasis. After induction dosing, it is administered every 12 weeks by SC injection. Tildrakizumab has demonstrated improved skin clearance compared with etanercept (Enbrel). Approval is expected by March 24, 2018.

Hepatitis C

Two new pan-genotypic (genotypes 1 through 6) hepatitis C virus (HCV) drugs are expected to be approved in August. Gilead’s voxilaprevir/sofosbuvir/velpatasvir is a single tablet that adds a protease inhibitor (voxilaprevir) to its sofosbuvir/velpatasvir (Epclusa) components. Sofosbuvir is a nucleotide analog polymerase inhibitor and velpatasvir is a nonstructural protein 5A (NS5A) inhibitor.

It is taken once daily for 12 weeks by patients with HCV who previously failed treatment with other direct-acting antivirals (DAAs). Approval is expected by August 8, 2017. AbbVie’s glecaprevir/pibrentasvir is a single tablet that contains a protease inhibitor (glecaprevir) and an NS5A inhibitor (pibrentasvir).

It is taken once daily for 8 weeks in treatment-naïve patients with HCV who do not have cirrhosis across all genotypes. For patients who have previously failed a DAA regimen, it is taken for 12 weeks. Approval is expected by August 19, 2017.

Cancer

The first CAR T immunotherapies are expected to gain FDA approval later this year. In CAR T therapy, some of the patient’s T cells are removed, modified to target the CD19 antigen expressed by certain cancer cells, and infused back into the patient. Novartis’ tisagenlecleucel-T is expected to be approved by September 29, 2017.

It is pending approval for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients. Kite Pharma’s axicabtagene ciloleucel is expected to be approved by November 29, 2017, to treat patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma who are not candidates for a stem-cell transplant.

Tisagenlecleucel-T and axicabtagene ciloleucel are breakthrough therapies that have demonstrated strong efficacy in these patient populations who have very limited treatment options.

Severe neurologic events and cytokine release syndrome (CRS) can be associated with CAR T therapies. CRS can cause high fevers, nausea, muscle pain, and, in some cases, low blood pressure and breathing difficulties. There are estimates that CAR T therapies may cost $300,000 or more for the 1-time infusion.