NasdaqGS:RAREBiotechs
Ultragenyx BLA Resubmission Puts UX111 And RARE Valuation In Focus
Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A.
The filing includes long term clinical data and responses to earlier regulatory concerns about the therapy.
If accepted, UX111 could become the first approved treatment specifically targeting this rare and serious condition.
Ultragenyx Pharmaceutical (NasdaqGS:RARE), trading at $24.07 as of the last close, is moving ahead with a key...