Congenital Lactic Acidosis (CLA)

September 18, 2017

Advicenne issued results of a phase III trial of ADV7103 in adults and children suffering from distal Renal Tubular Acidosis (dRTA). ADV7103 is given twice a day in contrast to the current standard of care (SoC), which are usually various unapproved products administered every four to six hours to attempt to re-balance the body’s pH and to normalize blood potassium level (kalaemia). The phase III study of ADV7103 was shown to restore the main biological defects observed, with the disease meeting positively primary and secondary endpoints. The objective of the study was to evaluate the efficacy, safety and acceptability of ADV7103, an innovative oral product, formulated in pediatric-friendly coated granules, that combines two active pharmaceutical ingredients. Normal blood bicarbonate levels were attained in most patients treated with doses of ADV7103 ranging from 0.75 to 8.45 mEq/kg/day. Mean doses of 1.7, 2.3, 3.8 and 6.1 mEq/kg/day ADV7103 were given, respectively, in adults, adolescents, children and infants. Non-inferiority of ADV7103 vs. SoC or baseline literature data was consistently demonstrated (per protocol, intention-to-treat, as well as sensitivity analyses). Kalaemia was normalized with ADV7103 with only two doses per day. These analyses were able to show that ADV7103 is superior to the SoC (p<0.0047).