FDA yesterday approved the combination of sodium phenylbutyrate and taurursodiol (Relyvrio, AMX0035, Amylyx Pharmaceuticals) as monotherapy or with existing approved treatments for the treatment of adults with amyotrophic lateral sclerosis (ALS). The drugs significantly slowed the loss of physical function in people living with ALS in a 24-week multicenter phase 2 clinical trial of 137 participants with ALS (CENTAUR) and an open-label 6-month extension trial. This new treatment for Lou Gehrig’s disease is taken orally by combining 1 packet in 8 ounces of water at room temperature. The recommended dosage for the first 3 weeks is 1 packet (sodium phenylbutyrate 3 g and taurursodiol 1 g) daily. After 3 weeks, the dosage increases to 1 packet twice a day. The medication can be taken before a snack or meal and can also be administered through a feeding tube. The approval of RELYVRIO is based on data from CENTAUR, encompassing a 6-month randomized, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase.
