Neurology News Network for the week ending August 22, 2020.
This week Neurology News Network covered the FDA approval of satrlizumab for patients with anti-aquaporin-4 antibody positive neuromyelitis optica spectrum disorder, results from a study evaluating the cost-effectiveness of epilepsy surgery, and the FDA approval of viltolarsen, the second FDA-approved therapy for patients with Duchenne muscular dystrophy amenable to exon 53 skipping.
Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.
Genentech has announced that the FDA has approved satralizumab for the treatment of adult patients with anti-aquaporin-4 antibody positive neuromyelitis optica spectrum disorder, only the second targeted treatment for this population and the first eligible for at-home administration. Marketed under the name Enspryng, the humanized monoclonal antibody targets and inhibits interleukin-6 receptor activity, which is thought to play a key role in the inflammation observed in NMOSD. Novel recycling technology allows for a longer duration of antibody circulation, translating into a subcutaneous dosing regimen of every 4 weeks after an initial loading dose. The approval pins satralizumab against the only other targeted treatment in this space, Alexion’s eculizumab (Soliris), which was approved with the same indication on June 27, 2019.
A study evaluating the cost of epilepsy surgery and surgical evaluation suggests that both the procedure and referral are cost-effective for eligible patients with drug-resistant temporal lobe epilepsy. Using a semi-Markov model to assess the cost-effectiveness of surgery and surgical evaluation over a lifetime horizon, investigators found that epilepsy surgery is cost effective compared to medical management in surgically eligible patients by virtue of being cost saving and more effective than management in the long run. The surgical evaluation for patients with DR-TLE remains cost-effective even if the probability of being deemed a surgical candidate as low as about 5%. Healthcare perspective costs showed that referral for surgery evaluation is estimated at $423,000 compared to $408,000 for those who continue medical management.
The US FDA has approved viltolarsen for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping — only the second FDA-approved therapy for this specific DMD gene mutation that affects 8% to 10% of patients with DMD. The agent from NS Pharma, delivered via weekly intravenous infusion, was granted accelerated approval via its priority review, fast track, orphan drug, and rare disease designations after its new drug application was accepted earlier this year. In March, NS Pharma launched an expanded access program for qualified patients. The approval was granted based on findings from a phase 2 clinical trial and long-term extension study, details of which were recently published in JAMA Neurology. Among 16 participants age 4 to 9, significant drug-induced dystrophin production was observed in both viltolarsen dose cohorts, with 15 (94%) patients achieving dystrophin levels greater than 2% of normal and 14 of 16 (88%) achieving levels greater than 3% of normal.
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