2017 U.S. drug approvals in neurology and psychiatry


The U.S. Food and Drug Administration approved 46 new therapies in 2017, up from only 22 the year before. The following is a list of the new medications for neurology and psychiatry.

  • Safinamide (Xadago, Newron Pharmaceuticals): a monoamine oxidase (MAO)-B inhibitor approved as adjunctive therapy in Parkinson’s disease with motor fluctuations. A phase III trial reported that safinamide 50 or 100 mg/day as an add-on to levodopa-carbidopa significantly increased On time and improved UPDRS Part III motor scores (Borgohain et al. Mov Disord 2014;29:229-237). The FDA rejected the drug in 2014, but approved the resubmission in March 2017.
  • Ocrelizumab (Ocrevus, Roche): an anti-CD20 monoclonal antibody for use in relapsing-remitting and primary-progressive MS. Approval was based on the OPERA I and II phase III trials in RRMS (Hauser et al. N Engl J Med 2017;376:221-234), and the ORATORIO trial in PPMS (Montalban et al. N Engl J Med 2017;376:209-220). The agent was touted as the biggest drug launch in 2017, with sales of USD$200 million in its first three months on the market. Ocrevus was subsequently approved for RRMS in Canada in August.
  • Deutetrabenazine (Austedo, Teva): this agent received two approvals – for chorea associated with Huntington’s disease, followed by an indication for tardive dyskinesia. In the Huntington’s study, mean total maximal chorea scores improved from 12.1 to 7.7 with deutetrabenazine vs. an improvement from 13.2 to 11.3 with placebo (Huntington Study Group, et al. JAMA 2016;316:40-50). Treatment success, as assessed with the Patient Global Impression of Change, was reported in 51% with active treatment vs. 20% with placebo. In the ARM-TD study, deutetrabenazine significantly reduced Abnormal Involuntary Movement Scale (AIMS) scores versus placebo (least-squares mean -3.0 vs. -1.6); treatment was associated with low rates of psychiatric adverse events, and no worsening of parkinsonism (Fernandez et al. Neurology 2017;88:2003-2010).
  • Valbenzazine (Ingrezza, Neurocrine): this agent, a vesicular monoamine transporter-2 inhibitor, was the first to receive approval for tardive dyskinesia, but only maintained its solo status for four months before Austedo was approved for TD (see above). Approval was based on the KINECT 3 trial, which showed a significant reduction in AIMS dyskinesia score with valbenzazine 80 mg/day versus placebo (least-squares mean change -3.2 vs. 0.1) (Hauser et al. Am J Psychiatry 2017;174:476-484). A separate phase II trial in Tourette’s syndrome was unsuccessful, but a new study is planned using a higher dose.
  • Cerlipnase alfa (Brineura, BioMarin): this agent, a recombinant human tripeptidyl peptidase-1 (TPP1), was approved to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a rare inherited neurodegenerative disease caused by a deficiency in TPP1. The drug will be one of the most expensive on the market, with a price tag of USD$702,000 per year before discounts, in part because of the low incidence of CLN2 (22 new patients in the U.S. per year).
  • Edaravone (Radicava, Mitsubishi Tanabe): this is the first drug approved for the treatment of amyotrophic lateral sclerosis in over 20 years. Although a phase III trial of edaravone failed in ALS, a post-hoc analysis suggested a benefit in a subgroup of patients with early-stage ALS who met certain criteria. A subsequent phase III trial reported a one-third reduction in the rate of disease progression in highly selected patients (Writing Group, et al. Lancet Neurol 2017;16:505-512).
  • Adjuvanted, recombinant varicella zoster vaccine (Shingrix, GlaxoSmithKline): while not a neurology/psychiatry drug, zoster vaccines are becoming routine in the treatment of MS patients scheduled for a course of immunosuppressants. The vaccine was approved for the prevention of shingles in adults >50 years. Of interest is that it is a non-live vaccine, in contrast to Zostavax, a live-attenuated zoster vaccine approved a decade ago. Shingrix was approved in Canada in October. The U.S. Centers for Disease Control Advisory Committee on Immunization Practices stated that Shingrix is the preferred vaccine for shingles prevention, and can be administered in adults who previously received Zostavax (www.cdc.gov/vaccines/vpd/shingles/public/index.html).

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