NEW YORK – The Institute for Clinical and Economic Review (ICER) on Wednesday issued its verdict on what price would prove cost-effective for Nanoscope Therapeutics' MCO-010 (sonpiretigene isteparvovec), an investigational gene therapy for advanced retinitis pigmentosa (RP) and severe vision loss.
ICER, a nonprofit that conducts independent value assessments of healthcare interventions, in a report said the one-time gene therapy could be cost-effective if priced between $67,400 and $101,300 for administration to one eye. "We have a number of uncertainties about the efficacy and durability of this therapy given the limited evidence to date, but the underlying approach is remarkable," ICER Chief Medical Officer David Rind said in a statement.
Retinitis pigmentosa is a group of rare and inherited retinal diseases that result in progressive vision loss due to the degeneration of photoreceptor cells. There's no cure for RP, though other companies like Coave Therapeutics, Ocugen, and Restore Vision are also studying gene therapy approaches.
Dallas-based Nanoscope previously said it intends to submit a biologics license application (BLA) with the US Food and Drug Administration seeking approval of MCO-010 in the first half of this year. MCO-010 aims to restore vision for patients with advanced RP by enabling non-photoreceptor cells in the retina to express the MCO protein, which is sensitive to light. Although RP can be caused by variants in dozens of genes, Nanoscope's MCO-010 is designed to treat RP in patients regardless of their genetic mutation.
ICER rated the available evidence on the gene therapy as "promising but inconclusive," citing uncertainty regarding long-term efficacy, possible harms, and durability.
In the randomized, controlled Phase IIb RESTORE trial, 27 participants received one of two doses in one eye or underwent a sham protocol. After one year and up to 100 weeks, treated patients on average had clinically meaningful improvements in best-corrected visual acuity in both the low-dose and high-dose study arms, compared to the control group.
However, the authors of the ICER report noted that since RP affects different aspects of vision over time, it's challenging to measure benefit across patients with any one functional measure. Additionally, there are secondary outcomes described in the RESTORE study protocol that haven't been publicly reported, which they said raises concerns about reporting bias.
"The evidence base for treatment efficacy consists solely of the RESTORE trial, a 27-participant RCT that has yet to be published nor details fully made publicly available," the authors wrote. "A single, small trial may not generalize to all types of RP. … Further information is needed to fully appraise the evidence."
Nearly all participants in the treatment groups experienced at least one mild or moderate ocular adverse event, such as intraocular inflammation or increased intraocular pressure, compared to just two-thirds of the control group. None of the participants had a serious adverse event and there were no deaths observed in the clinical trial.
ICER's price range of $67,400 to $101,300 assumes the treatment effects will remain for five years and that the gene therapy will be only administered to one eye, since the benefits of administering it to both eyes is unknown.
The report will be reviewed at a public meeting of one of ICER's independent evidence appraisal committees, the New England Comparative Effectiveness Public Advisory Council, in April.