Second historic Niemann-Pick disease nod from US FDA

People affected Niemann-Pick disease, a rare and fatal genetic disorder, have waited a lifetime for an effective medicine. Now, in the space of a week, the US regulator has approved two.

The Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine), developed by privately-held rare disease specialist IntraBio, for treating neurological symptoms associated with Niemann-Pick disease type C (NPC).

NPC is one of a group of metabolic disorders where harmful quantities of fatty substances accumulate in the body. The condition is progressive, and patients face increasing neurological impairment and loss of motor function.