Posted by Michael Wonder on 19 Jan 2023
FDA grants rare paediatric disease status to GC Pharma’s San Filippo syndrome therapy
18 January 2023 - GC Pharma said on Wednesday that its San Filippo Syndrome type A treatment, jointly being developed with bio-venture Novel Pharma, has been designated as a rare paediatric disease by the US FDA.
The two companies have been jointly developing enzyme replacement therapy for the disease since 2020.
