Shire picks up two breakthrough badges for rare disease drugs
14 June 2016

US regulators have awarded 'Breakthrough Therapy' badges to two investigational products being developed by Shire: a novel formulation of budesonide for eosinophilic esophagitis and maralixibat for

Marathon Files NDA for Duchenne Drug
14 June 2016

Marathon Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for the glucocorticoid deflazacort to treat Duchenne muscular dystrophy (DMD) patients.

Catalyst Gets FDA Nod to Conduct Confirmatory LEMS Study
13 June 2016

Five months after Catalyst Pharmaceuticals received a Refusal to File letter from the FDA, the 2 parties have agreed on the study design for the confirmatory Phase 3 study that the company needs to

Novartis’ Jakavi comes out on top in rare blood cancer trial
13 June 2016

Novartis has presented clinical data showing that its Jakavi was superior to best available therapy in patients with the rare haematological cancer polycythemia vera (PV), which affects around 100,

New AML Drug Shows Promise in Phase I Trial
13 June 2016

New Data From uniQure's Gene Therapy for Hemophilia B is Impressive
12 June 2016

At the 21st Congress of the European Hematology Association (EHA) in Copenhagen, Denmark this weekend, data from a phase 1/2  trial looking at the safety and efficacy of uniQure’s

Trifarotene for Lamellar Ichthyosis Performs Well in Phase I Study
11 June 2016

Drug developer Galderma announced their treatment, trifarotene, was safe and well-tolerated in its phase I study.

MS therapy could prevent relapses long-term, study indicates
10 June 2016