JD Supra

New FDA approval process promotes development of rare disease gene therapies

The U.S. Food and Drug Administration (FDA) announced a new process called “Rare Disease Evidence Principles” (RDEP), under which eligible drugs and biologics for ultra-rare diseases caused by known ...
RAPS

Investment in quality management can reduce defects and recalls, FDA says

Companies that invest in quality management initiatives can incur lower costs related to defects, waste, and recalls compared to those that do not make such investments, according to a white paper ...
JD Supra

FDA’s Rare Disease Evidence Principles Process Provides Guidance for Applications Supported by a Single Pivotal Trial

On September 3, 2025, the US Food and Drug Administration (FDA) announced a new process to support the development of drugs intended to treat rare genetic diseases. The Rare Disease Evidence ...
RAPS

FDA warning letters address CGMP, clinical study plan failures

Intas Pharmaceuticals was again in hot water with the US Food and Drug Administration (FDA) for a host of current good manufacturing practice (CGMP) violations, including manipulating particle counts ...