The candidate will now benefit from the incentives given to those drugs granted orphan status through the FDA’s ODD programme, introduced by the organisation to improve the availability of treatments for rare diseases.
FSHD is characterised by the life-long, progressive loss of muscle function that typically causes weakness in muscles in the face, shoulders, arms and trunk, before progressing to muscles in the lower body.
Avidity’s AOC 1020 is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4.
