US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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Creating a New Therapy

MDA is often asked why it takes so long to create a new therapy — after all, we are constantly funding, and reporting on, exciting new research, and mouse “cures” seem common. However, a lot of work goes into the time between discovering that a drug works in a mouse and testing that drug in a clinical trial, and then to getting a therapy to patients. This is not “lost” time. It is time spent ensuring that the drug has the best possible chance of having an effect on the disease and the lowest possible chance of causing harm. The first steps are termed “preclinical research” and encompass an array of steps. Even the most efficient and lucky of drug developers take a minimum of two years to get through this process.

Although frustrating, these steps are necessary to getting a safe and effective drug to people with neuromuscular diseases. The developers must do toxicology studies to show the U.S. Food and Drug Administration (FDA) that the drug is relatively safe; they must show that the drug is likely to be effective; and they must determine how to administer the drug and how much to give.

On average, preclinical research takes five to seven years and costs several million dollars to complete, according to FasterCures (a nonprofit organization dedicated to increasing the speed of drug discovery across all therapeutic areas). Once this work is done, the drug developer can apply to the FDA for approval to start clinical trials by submitting an investigational new drug application (IND). If the IND is approved, the developer is cleared to start clinical trials.

These trials take another five to seven years to complete (and many more millions of dollars) before the developer can ask the FDA for approval to bring a drug to market.

What is going on during all this time?

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