Biogen reverses course on compassionate use for ALS drug tofersen with plans to open program in July
Despite immense pressure from the ALS community, Biogen had repeatedly refused to open investigational drug tofersen for use outside of clinical trials. But now, caving to that demand, Biogen has reversed its stance.
With a pivotal Phase III study in ALS ongoing and data expected to read out later this year, Biogen just a month ago said it wouldn’t offer tofersen for compassionate use in an attempt to be fair to patients on placebo in that late-stage test. The original goal was to wait for top-line efficacy and then open an Early Access Program for patients with a specific mutant variation of the SOD1 gene that comes with a particularly poor prognosis.
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