PepGen: Q2 Newsletter

PepGen has released their Q2 Newsletter, including an update for the Canadian Duchenne muscular dystrophy community regarding the CONNECT1-ED051 clinical trial.

Italfarmaco has completed the FDA submission of New Drug Application (NDA) for Givinostat in Duchenne muscular dystrophy and receives priority review.

Edgewise Therapeutics has announced positive 12-month topline results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD).

The US Food and Drug Administration (FDA) has approved ELEVIDYS, the first gene therapy for Duchenne muscular dystrophy developed in partnership by Sarepta Therapeutics and Roche.

Santhera has granted an exclusive North America license for vamorolone in the treatment of Duchenne muscular dystrophy to Catalyst Pharmaceuticals.

NS Pharma has received FDA clearance to initiate a Phase I/II study for NS-050/NCNP-03, an exon 50 skipping candidate for the treatment of Duchenne muscular dystrophy.

FibroGen has announced topline data from the Phase 3 LELANTOS-1 study of pamrevlumab, their investigational monoclonal antibody, for non-ambulatory patients with Duchenne muscular dystrophy.

PepGen has received clearance by Health Canada for the CONNECT1-EDO51 Phase 2 clinical trial of PNG-EDO51, an exon 51 skipping therapy.

The first-ever FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne muscular dystrophy received a positive vote for accelerated approval of SRP-9001.

Santhera has shared it’s launch plans for vamorolone in the US, EU, and UK, as well as an update on the FDA mid-cycle review.