The Food and Drug Administration’s independent panel of experts decided on Wednesday against Biogen’s ALS treatment efficacy.
ALS medication is under consideration for a rare and aggressive type of illness. Tofersen is a medication that was created to treat a rare hereditary variant of amyotrophic lateral sclerosis or ALS. Three advisors supported effectiveness, five opposed it, and one abstained.
Clinical Trial VALOR
Data from the Phase 1/2/3 clinical trial VALOR support Biogen’s application for accelerated approval of tofersen. (NCT02623699). The Phase 1 and 2 portions of the trial demonstrated that tofersen could lower SOD1 protein levels as intended, as well as that the medication was typically well tolerated.
108 persons with ALS were randomly randomized to receive eight injections of tofersen (100 mg) or a placebo over the period of 28 weeks, or about six months, in the Phase 3 portion of VALOR. The primary purpose of the trial was to evaluate the effect of medication on disease progression as determined by the ALS Functional Rating Scale-Revised. (ALSFRS-R).
Newer data from an open-label extension study (NCT03070119), in which 95 VALOR patients are being treated with tofersen for up to seven years, showed that disease progression was slower among patients who received tofersen in the initial study, compared to those who received a placebo for six months in the original trial.
VALOR analysis revealed that tofersen-treated patients with a greater NfL drop in the early months of treatment tended to have slower disease progression.
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Seeking Expedited ALS Treatment Approval
Tofersen is being sought under the FDA’s expedited approval pathway, which allows the agency to grant conditional marketing authorization for medicines where preliminary biomarker findings indicate a potential benefit.
Developers of accelerated-approval medicines are required to do further studies to confirm a clinical benefit. According to Biogen and Ionis, the committee’s unanimous vote likely supports speedy approval.
Because NfL is a common sign of nerve injury and is raised in a variety of neurological illnesses, using it as a viable surrogate biomarker of efficacy may have ramifications beyond ALS. It remains to be seen whether the FDA will consider the NfL evidence sufficient to sustain an accelerated approval.
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