Regenxbio Inc. (NASDAQ:RGNX) on Monday said it aligned with the Food and Drug Administration (FDA) regarding the next steps needed for a potential accelerated approval of Navsunli (RGX-121, clemidsogene lanparvovec).
The potential one-time gene therapy is seeking approval for Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.
The Wall Street Journal was the first to report it, highlighting another policy shift at the agency under its new leadership.
FDA Reverses Rejection Of Regenxbio’s Gene Therapy
The FDA acknowledged that the existing Navsunli data are sufficient to be considered for the accelerated approval pathway and that the company does not need to enroll additional patients or conduct additional studies, including the FDA’s previously recommended incorporation of an untreated control arm.
