Downfall of BioMarin's Kyndrisa raises more doubts on Sarepta's eteplirsen

Rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) has withdrawn its European Marketing Authorization Application (MAA) for Kyndrisa (drisapersen), an experimental drug for Duchenne muscular dystrophy (DMD).

During discussions with the US-based biotech, the European Medicines Agency’s Committee for Medicinal Products for Human Use had indicated that it intended to issue a negative opinion.

Based on discussions at the CHMP meeting and the US Food and Drug Administration's Complete Response Letter in January, BioMarin intends to discontinue clinical and regulatory development of Kyndrisa.