Rare disease spotlight: a leukodystrophy with small pipeline behind first approval

As U.S. patients with metachromatic leukodystrophy begin to get access to the first approved therapy for the rare disease, the pipeline coming up behind Orchard’s Lenmeldy is shrinking.

At least three programs were recently discontinued for metachromatic leukodystrophy (MLD), reducing the disclosed pipeline by half. Two of the three remaining companies still developing a program for the indication are preclinical, while the one clinical company is seeking out-licensing opportunities for its Phase I/II asset. ...