nusinersen-treated group. Over the course of the
study, 31% of the nusinersen-treated infants required
permanent ventilation, defined as at least 16 hours per
day, compared with 48% of the control infants.
The ENDEAR study was supported by Ionis Pharmaceuticals and Biogen.
Good News, Bad News
Following Dr. Kuntz’s plenary presentation of the
ENDEAR study results, Charlotte J. Sumner, MD,
Associate Professor of Neurology at Johns Hopkins
University in Baltimore, served as the discussant.
While Dr. Sumner praised the study findings and the
breakthrough they represent, she did point out the
staggering cost of the drug. At about $120,000 per
dose, the price “has raised issues about insurance ap-
proval and reimbursement and raises concerns about
delays to treatment initiation and institutional risk,”
she said. “But I would say that despite these chal-
lenges, well over 100 patients have already been dosed
commercially at very different ages, and this is very
promising that we will be able to deliver this drug in a
widespread way.” NR
—Glenn S. Williams
Finkel RS, Chiriboga CA, Vajsar J, et al. Treatment of infantile-onset spinal
muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation
study. Lancet. 2016;388(10063):3017-3026.
This one you can not miss
45th Annual Meeting
Southern Clinical Neurological Society
January 12th - 19th 2018
Save the Dates