Denali Therapeutics Announces FDA Has Selected DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo Syndrome Type A) for START Pilot Program Intended to Accelerate Development of Rare Disease Therapies
DNL126 is an investigational enzyme replacement therapy designed to cross the BBB for the potential treatment of MPS IIIA (Sanfilippo syndrome type A).
- DNL126 is an investigational enzyme replacement therapy designed to cross the BBB for the potential treatment of MPS IIIA (Sanfilippo syndrome type A).
- Selected participants of the START Pilot Program are provided opportunities to obtain frequent advice and engage in more rapid ad hoc communication with FDA review staff to address product-specific development issues.
- Given the advanced development stage of the program, Denali did not apply to START for tividenofusp alfa.
- The FDA granted Fast Track designation to tividenofusp alfa, which also facilitates increased communication and engagement with the FDA specific to this program.