How Drugs Are Developed
A long journey from the lab to a patient
A long journey from the lab to a patient
The development of a new drug is a challenging and time-consuming process. Even once an investigational product begins testing in humans, it can take more than 10 years for a new drug to gain approval by the U.S. Food and Drug Administration (FDA) for use in patients.
Sarepta’s approach to creating an investigational product builds efficiencies into research and development. However, all new products have to follow the same steps before they can be studied in people. We are working with a sense of urgency to develop products to treat progressive genetic conditions that affect infants, children, and adults.
A drug that is not yet approved by the FDA (or other health authority outside the U.S.) and is still being studied. It must go through clinical trials in humans first to determine if the drug is safe and effective.
Discovering new possibilities
The process of research and testing in animals—referred to as the pre‑clinical phase—must be completed before an investigational product moves to the clinical phase, in which drugs are tested in people.
Studies performed in the lab and in animals before any testing in humans is done.
PRE-CLINICAL PHASE
Step 1: Discovery
Scientists discover a new compound with the potential to become an FDA‑approved medicine.
Step 2: Testing
The drug company tests the drug in the laboratory, then advances to animal studies to learn more about its safety and how it works.
Step 3: IND Application
The drug company submits an Investigational New Drug (IND) application to the FDA with key information, including a description of what the drug is made of and how it will be manufactured, the results of animal testing, and plans for how the drug will be tested in humans (clinical trials).
Step 4: IND Review
The FDA works to ensure proposed clinical trials do not place participants at extreme risk or harm and provides informed consent and protection for participants.
After the FDA reviews and approves the IND, clinical trials may begin.
The unique challenges in rare genetic conditions
There are unique challenges when designing clinical studies for rare genetic conditions. These include small numbers of people diagnosed with a certain illness, different signs and symptoms among individuals, and different rates of disease progression.
Small patient populations make it challenging to recruit for clinical trials, while a condition’s heterogeneity can make it hard to define uniform, meaningful measurements of results. It is challenging to develop investigational products when there is a lot of variability in disease presentation, severity, or progression within the small population being studied, and very few measurable or proven ways to understand what the impact of treatment will be.
Clinical trials in rare conditions present unique challenges to researchers.
Variations in risk factors, symptoms, and presentation of a condition’s characteristics between individuals with the same condition.
Explore the Sarepta pipeline of products
Sarepta has more than 40 research programs in different stages of development. Many are still in the pre-clinical stage, and others are moving through the various phases of clinical trials. Each investigational product must move through all stages of discovery, investigation, and clinical trials, and must receive FDA approval before it can be made available to patients.
Sarepta is investigating gene therapy, RNA technologies, and gene editing as potential approaches to treat rare genetic conditions. We are exploring new investigational products for neuromuscular, central nervous system, and cardiac diseases.
Ribonucleic acid, a transcript of information from DNA that has several functions, many of which relate to making proteins.
