Different Applications For Small And Large Molecules

Q.1 Compare and contrast NDA and BLA. Why are there different applications for small and large molecules? A small molecule drug approval required a New Drug Application (NDA) because it covered under Federal Food Drug and cosmetic Act Section 505 while large molecule drug required a Biologics License Application (BLA) because it covered under Public Health Service Act Section 351. Compared with conventional small-molecule drugs, products derived from a biological source are structurally complex, large molecules and involved in different manufacturing processes that require tight control to ensure their safety, purity, quality, and efficacy. Because of these differences in respect of the product characteristics and manufacturing process,†¦show more content†¦The regulations regarding BLAs for therapeutic biological products included in 21 CFR parts 600, 601 and 610. Form 356h is a harmonized form, and a sponsor can use it for NDA, BLA and Abbreviated New Drug Application (ANDA). Drugs are nominated as orphan drugs for those diseases with patient population of less than 200,000 in the US. The FDA has given special provision for the development, marketing approval and marketing of orphan drugs under 21 CFR Part 316.The Orphan Drug Act provides incentives to organizations to research and test drugs because it have limited commercial returns due to the small size of the patient group. Companies are taking commercial risks so FDA is providing assistance in the forms of NDA fee waivers, tax credits for clinical research, and grants for the research. The FDA also provides market monopoly to the organization to market the drug for seven years. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups. The Orphan Grants Program from FDA has been used to bring more than 45 products in the market. 21CFR 316 gives FDA right to revoke orp han drug designation if they found that untrue statement regarding material fact or drug is not eligible as an orphan drug and withdraw marketing monopoly.