RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Key Takeaways The FDA granted Breakthrough Therapy designation to Ultragenyx's GTX-102 for Angelman syndrome. GTX-102 showed rapid, lasting improvements in AS patients during a phase I/II study over up to 3 years. RARE is enrolling for a phase III Aspire study and planning a broader phase II/III Aurora study for GTX-102.Ultragenyx Pharmaceuticals (RARE) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), t ...