Japan’s Ministry of Health, Labor, and Welfare (MHLW) granted orphan drug designations to 15 drug compounds on February 28, including Novartis Pharma’s atrasentan for IgA nephropathy. Other drugs given this designation include Alexion’s efzimfotase alfa (hypophosphatasia), HanAll Biopharma’s batoclimab (thyroid eye disease), SpringWorks Therapeutics’ nirogacestat (desmoid tumors), and Nobelpharma’s naxitamab (neuroblastoma), among others. Eight of these orphan drugs qualify for priority review, including eneboparatide, nirogacestat, and retifanlimab. However, even though Japan recently relaxed its requirements for orphan drug designation, a reduced workforce at the Pharmaceuticals and Medical Devices Agency (PMDA) means that priority review remains restricted to drug products meeting previous criteria – that are more stringent.
India’s Central Drugs Standard Control Organisation (CDSCO) has broadened the scope of its surprise inspections beyond drug companies to include medical device manufacturers, drug testing laboratories, and contract research organizations (CRO). With these Risk-Based Inspections, the CDSCO aims to bolster regulatory confidence and enhance compliance across the healthcare sector. This initiative follows the success of over 400 surprise inspections in the pharmaceutical sector during the last 2 years – which have significantly reduced the number of complaints and low-quality drug products. Regulators and teams of experts have begun inspections at several other medical locations with a focus on assessing data integrity.
Written by: Ames Gross – President and Founder, Pacific Bridge Medical (PBM)
Mr. Gross founded PBM in 1988 and has helped hundreds of medical companies with regulatory and business development issues in Asia. He is recognized nationally and internationally as a leader in the Asian medical markets. Mr. Gross has a BA degree, Phi Beta Kappa, from the University of Pennsylvania and an MBA from Columbia University.