The Institute for Clinical and Economic Review (ICER) will compare the clinical effectiveness and value of the only approved disease-modifying treatment for spinal muscular atrophy (SMA) — Spinraza (nusinersen, by Biogen) — and an investigational gene therapy showing promise in advanced clinical trials — Novartis‘ AVXS-101, the nonprofit analysis group announced in a press release.
Those interested in participating in this report can, through Aug. 17, give information and viewpoints to ICER: an “Open Input” period is now underway to collect relevant information.
ICER also intends contact key patient and advocacy groups, as well as clinical specialists, to better understand the perspectives of patients, caregivers, and health professionals on both Spinraza and AVXS-101.
A potential one-time SMA gene therapy, AVXS-101 was developed and moved into advanced clinical testing by AveXis, which was acquired by Novartis this year. Spinraza, approved to treat both children and adults with almost all SMA types, is administered through a series of injections into the spinal canal.
ICER’s report will be reviewed at a March 2019 meeting of the New England Comparative Effectiveness Public Advisory Council, one of the Institute’s three independent evidence appraisal committees.
The research institute, which produces reports analyzing evidence on the effectiveness and value of treatments, offers patient groups and manufacturers more detailed information about its public engagement process in its Manufacturer Engagement Guide, the Patient Participation Guide, and the Patient Guide to Open Input. These documents explain the types of information considered most useful in compiling reports.
Information supplied has no limit as to its length, and can be sent by email to ICER through Aug. 17 at 5 p.m. EST.
About a week later, around Aug. 22, ICER plans to release a Draft Scoping Document with details on the proposed scope of its evidence review. This document will be open for public comment until Sept. 7.
AVXS-101 is designed to deliver by intravenous injection a functional copy of the SMN1 gene — which causes SMA when it is unable to produce a working SMN protein — directly to patients’ motor neurons, nerve cells that control muscle contraction.
According to the press release, analysts expect the gene therapy to be approved by the U.S. Food and Drug Administration in early 2019.
AVXS-101 is being evaluated in clinical trials that include the SPR1NT Phase 3 study (NCT03505099) in presymptomatic infants younger than 6 weeks (42 days) and diagnosed with SMA types 1-3; the STR1VE Phase 3 trial (NCT03306277) in SMA type 1 children younger than six months, and its European equivalent STR1VE-EU (NCT03461289); and the STRONG Phase 1 trial (NCT03381729) in type 2 children up to age 5 (60 months old).
Three of these trials are reported to still be recruiting patients; STR1VE is not. More information can be found by clicking on each trial’s NCT numbers, given above.
Spinraza became the first disease-modifying therapy to be approved by the U.S. Food and Drug Administration (FDA) to treat SMA types 1-3 in children and adults in December 2016, based on data showing significant improvements in both motor function and survival in two Phase 3 clinical trials, ENDEAR (NCT02193074) and CHERISH (NCT02292537).The European Commission followed in 2017, approving Spinraza to treat patients with types 1_3.
Spinraza is designed to boost the ability of the SMN2 gene to produce a full-length and functional SMN protein. Without Spinraza, most of the protein generated from SMN2 is unstable and shorter than usual, and as such cannot compensate for the lack of a healthy SMN1 gene.