Five months after Sanofi backed away from a proposed deal with Maze Therapeutics on a Pompe disease therapy after an antitrust challenge, Shionogi has snapped up the drug.
Signs that the Federal Trade Commission (FTC) in the US may be softening its stance on pharma deals seem to have been misread, as the regulator has now sued to block Sanof
Sanofi is already a big player in the Pompe disease market with injectable drugs like Nexviazyme and Myozyme/Lumizyme, but has now set its sights on an oral drug candidate
Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe
Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines
The safety of Astellas' gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845.
Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51.
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.