Accelerating the Development of Orphan Drugs for Rare Diseases

USP’s Sheehan and Liu talk at AAPS PharmSci 360 abou getting novel excipients past the monograph hurdle through early engagement with FDA.

CDMOs announced expansions, exhibitors showcased automated equipment, and supply chain strategy discussions were a focus at CPHI Milan 2026.

Mitigating nitrosamine risk requires assessing each product individually and chasing zero nitrite in pharmaceutical excipients is often impractical and costly.

The business case for regulated AI turns on the cost of producing an outcome that the quality system can accept.

In October, FDA reorganized rare disease development around a fit-for-purpose framework and a new pathway for individualized gene editing. Regulators in Europe and the UK set out long-term plans for clinical trials, AI, and moving away from animal testing.

Clinical, manufacturing, safety, and commercial teams all lean on shared data. They need to agree on who publishes each data product, who can use it, and what evidence goes with it.

Structural modifications can improve peptide stability, solubility, and absorption to enable effective oral delivery.

The FDA approves VOCERYQ, the first DNA medicine, for RRP surgery reduction, raising plasmid, drug-device, and confirmatory trial manufacturing demands.