Shots:The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…
INTRODUCTIONDuchenne Muscular Dystrophy (DMD) is a rare and the most frequent genetic neuromuscular disorder affecting all races and ethnicities (affecting approximately 1 in 3,500 population). It is characterized by progressive muscle weakness and some specific abnormalities like changes in muscle fiber size, scar tissue formation, muscle fiber necrosis, and inflammation evidenced from muscle biopsy…
The US FDA has approved multiple NDAs and BLAs in Aug 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 69 novel products so far in 2020, including 10 in Aug 2020. Additionally,…

