New and promising data from AveXis’s SMA gene therapy

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,p class=”subgreen”>New data from AveXis’s AAV vector based gene therapy shows promise as the spinal muscular atrophy (SMA) drug improves on previous results from eight months prior.

AveXis released this latest data in the New England Journal of Medicine which details improvements in subjects’ ability to speak and sit up unassisted. Of the 12 patients in the study, 11 are now able to speak – 3 up from the previous 8. Another two patients also gained the ability to sit unassisted bringing the total to 9 of the 12. The number of patients in the trial who do not need permanent ventilation is also encouraging. These are positive results from the company whose therapy would rival Biogen’s Spinraza if it gains market approval.

Questions that remain for the company include how to price the therapy, particularly given Spinraza’s substantial $750,000 cost; and the possibility of the therapeutics effects fading and thus creating a need for readministration.

AveXis CEO Sean Nolan commented to investors, ‘Both of the regulatory agencies have agreed that the … confirmatory trials for SMA Type 1 would be a single arm utilizing natural history as the control with relatively few patients, 15 or 20 in the U.S. and approximately 30 in EU. And they have each asked us to seek discussion with them to talk about accelerated approval or conditional approval.’

Source: Chasing Biogen, AveXis posts improved gene therapy data Press Release