Tiny biotech’s experience raises questions about FDA’s rare disease policies

The experience of Grace Science, a biotech developing a gene therapy for an ultrarare, fatal condition, shows how gaps between FDA’s commitments to accelerating rare disease drug development and the reality experienced by struggling biotechs impact families facing serious diseases.

According to Grace Science LLC, despite early signs of efficacy for a therapy addressing a well-characterized monogenic disorder, NGLY1 deficiency, FDA told the company it does not qualify for the plausible mechanism framework and must complete a new manufacturing run before seeking approval. ...