FDA grants orphan drug designation to Concert’s cystic fibrosis treatment
Last month, the company had begun a phase 2 trial in the US for studying CTP-656 in patients with cystic fibrosis and having gating mutations. The phase 2 trial’s
Arna Pharma and Slate Run Pharmaceuticals have completed a joint venture (JV) to form a speciality pharmaceutical company in the US focusing on branded products, 505(b)(2) medicines, and specialised generics.
Gilead Research and Development executive vice president and chief scientific officer Norbert Bischofberger said: “Direct-acting antiviral treatments have transformed our ability to treat hepatitis C; however, for some
Abeona Therapeutics president & CEO Timothy Miller said: “This designation builds on our commercial portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an