FDA maps approval path for one-patient gene editing drugs

The FDA has described how a company can seek approval for a gene editing treatment built for one patient. Endpoints News reported the outline on October 28. STAT reported the draft rules a day earlier. They would let companies update personalized therapies without filing a new biologics license application. STAT says the framework is called an “individualized feature space.”

Gene editing changes a person’s DNA to fix a faulty gene. A treatment built for one person’s exact mutation doesn’t fit the usual drug rules. Those rules lean on large trials. A mutation only one person has can’t fill a large trial. The FDA’s February draft guidance, called the plausible mechanism framework, uses a different test. A company has to identify the disease-causing abnormality. It must show the therapy targets the root cause. It must also confirm the edit worked on its target. The FDA also points to natural history data from untreated patients. For now, Fierce Biotech reports, the pathway covers genome editing and RNA-based methods. Those are drugs that target the underlying cause of a rare disease.