BRIEF—Clinigen and Santhera launch US Expanded Access Program with idebenone

Pharma services firm Clinigen Group has initiated an Expanded Access Program (EAP) in the USA with Switzerland-based Santhera Pharmaceuticals for idebenone in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline.

The EAP (also known as a Managed Access Program), referred to as BreatheDMD, provides a mechanism through which eligible patients in the USA with DMD can obtain access to investigational idebenone, through a network of research centers across the USA.

Santhera announced plans for the program earlier this month, but not the partnership with Clinigen.

Idebenone is a medicine that is currently under investigation for the treatment of DMD in patients who are in respiratory decline. The firm’s request for expedited regulatory treatment with Raxone/Catena (idebenone) in the USA was rejected by the FDA in late 2016. Last month Santhera said it anticipated a negative opinion for Raxone from the European Medicines Agency.