How are you counseling your patients with ALS taking sodium phenylbutyrate and taurursodiol on continuing treatment in light of the negative phase 3 trial results?
Answer from: at Academic Institution
AMX0035 also known as phenylbutyrate and taurursodiol received accelerated approval from the FDA in 9/2022 following a phase II trial demonstrating a preliminary signal of efficacy. A larger phase III placebo-controlled trial (PHOENIX trial) with >600 subjects was recently completed and, unfortun...
I would continue the medication if patients who have been taking it have found it beneficial and want to continue it, even though trials did not confirm that it was as effective as it had been previously reported. There is room for some degree of doubt and there is no known danger. Previous patients...