Australia: Orphan Drug Designation For Kalydeco + Lumacaftor For Cystic Fibrosis

Orphan Druganaut Blog

Australia’s Therapeutic Goods Administration (TGA) on September 22nd, designates Lumacaftor (VX-809) as an orphan drug in combination with Kalydeco (Ivacaftor), for the treatment of Cystic Fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene.

The combination of Lumacaftor + Kalydeco has the following Orphan Drug Designations:

•   FDA ODD in June 2014

•   EMA COMP recommendation in July 2014.

At the upcoming North American Cystic Fibrosis Conference (NACFC), October 9-11 in Atlanta, Vertex Pharmaceuticals will present the following Abstracts on the Phase III TRAFFIC and TRANSPORT studies of Lumacaftor in combination with Kalydeco (Ivacaftor), in CF patients with two copies of the F508del mutation:

•   “Effect of Lumacaftor in combination with Ivacaftor in patients with cystic fibrosis who are homozygous for F508del-CFTR: Phase 3 TRAFFIC & TRANSPORT studies.” An oral presentation of these data will be delivered as…

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