Payer discomfort with accelerated approvals exploded into the public arena with the Medicare national coverage determination on Aduhelm and similar drugs, and developers worry that the fallout will be far reaching.

Payer discomfort with accelerated approvals exploded into the public arena with the Medicare national coverage determination on Aduhelm and similar drugs, and developers worry that the fallout will be far reaching.

Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.

Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."