BRIEF—Abionyx treats rare disease patients under compassionate use rules

French biotech Abionyx Pharma will treat a fourth patient with its rare kidney disease candidate CER-001, following a compassionate access authorization (CAA).

CER-001, a negatively-charged lipoprotein particle containing human recombinant apoA-I, is a candidate for treating LCAT deficiency, a severe and fatal disease for which there are no treatments.

Abionyx said “constructive discussions have been initiated with the European regulatory authorities to define a pathway for the rapid advancement” of the candidate in this indication.

The company also announced  positive mid-stage data for CER-001 in January, for the treatment of septic patients at high risk of developing acute kidney injury (AKI).