Tag: orphan disease

FDA approves Marathon Pharmaceuticals’s orphan drug
February 9, 2017 Off

FDA approves Marathon Pharmaceuticals’s orphan drug

By Dino Mustafić

The U.S. Food and Drug Administration (FDA) has approved Marathon Pharmaceuticals’s Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.

FDA grants ODD to GenSight’s retinitis pigmentosa drug
January 31, 2017 Off

FDA grants ODD to GenSight’s retinitis pigmentosa drug

By Dino Mustafić

GenSight Biologics, a biopharma company that discovers and develops innovative gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the Company’s product candidate GS030 for the treatment of retinitis pigmentosa.