FDA approves first gene therapy for Sanfilippo syndrome type A - Pharmaceutical Technology

With a $3.95m price tag, Fayuvi – a one-time gene therapy – is one of the most expensive drugs on the market. With a $3.95m price tag, Fayuvi – a one-time gene therapy – is one of the most expensive drugs on the market. While current drugs and medical devices help manage daily symptoms, Ultragenyx’s gene therapy is the first disease-modifying treatment approved for patients. Credit: Michael Vi/Shutterstock.com. The US Food and Drug Administration (FDA) has approved Ultragenyx’s gene therapy for Sanfilippo syndrome type A, marking the first treatment of its kind greenlit for children with the genetic neurodegenerative disorder.…

Source: Pharmaceutical Technology