Ultragenyx Pharmaceuticals' apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The failure of what was once considered a potential blockbuster therapy is forcing Ultragenyx to examine significant cost cuts.
The New England Journal of Medicine retracted the main clinical trial used to approve Amgen's $500 million drug Tavneos, following allegations of data manipulation. The European Medicines Agency had previously recommended revoking the drug's approval over data integrity concerns.
The FDA has rejected RP1, a breakthrough melanoma treatment, twice despite its promise in treating skin cancer. The rejections have left medical professionals puzzled and concerned about implications for future drug approvals.
