Denali Therapeutics Scores FDA Approval for Rare Disease Treatment, Boosts Regenxbio

Denali Therapeutics has received FDA approval for the first new drug in 20 years to treat a rare genetic disease that affects cognitive abilities in children. The approval comes ahead of schedule and is a month after the FDA rejected a gene therapy from Regenxbio. Denali's drug, ZYNTEGLO, is expected to significantly increase the production of a protein that is deficient in patients with Hunter syndrome. The approval is a significant milestone for the company and the treatment of this rare disease.