FDA’s accelerated approval messaging collides with CRL reality

FDA’s complete response letter for Regenxbio’s clemidsogene lanparvovec gene therapy for Hunter syndrome (MPS II) is the latest in a series of agency decisions that have stalled or killed drug development programs for debilitating diseases. Many of the setbacks involve applications for accelerated approval for an orphan condition, but some drugs for high-prevalence diseases have also been affected.

More CRLs for orphan accelerated approval applications are expected in the coming months, FDA officials and company representatives with direct knowledge of the agency’s deliberations told BioCentury. ...