EMA removes negative note on label of PTC's Duchenne drug; GSK, Akebia win Japan OK for oral anemia drugs
→ PTC Therapeutics $PTCT won conditional approval to market ataluren for Duchenne MD in Europe way back in 2014 — despite the fact that all the data pointed to a failure. And it’s operated with a label saying that efficacy had never been proven for a group of patients. Now the CHMP recommended by a majority of votes to remove the statement “efficacy has not been demonstrated in non-ambulatory patients” from the SmPC for Translarna. “We are excited to see that the CHMP adopted the positive opinion for this label modification allowing patients who become non-ambulatory to continue to use Translarna,” said CEO Stuart Peltz.
→ Japan has approved a pair of oral hypoxia-inducible factor prolyl hydroxylase inhibitors to treat anemia due to chronic kidney disease. It’s the first approval anywhere in the world for both GlaxoSmithKline’s daprodustat (now Duvroq) and Akebia’s vadadustat (now Vafseo). Kyowa Kirin is in charge of marketing for GSK while Akebia has partnered up with Mitsubishi Tanabe Pharma Corporation on commercialization. Both drugs work by mimicking the physiologic effect of altitude on oxygen availability: stabilizing hypoxia-inducible factors, which lead to increased red blood cell production, iron metabolism and oxygen delivery to tissues.