Pipeline
Amicus Therapeutics is developing a diverse pipeline of novel, small molecule, orally-administered pharmacological chaperones to treat a range of human genetic diseases. Amicus has completed multiple Phase 2 studies of its lead product, AT1001, for Fabry disease and is currently conducting Phase 2 studies of AT2101 for Gaucher disease. Phase 1 clinical studies of AT2220 for Pompe disease were completed in October 2007. Amicus is leveraging its core pharmacological chaperone platform to actively pursue therapies in other genetic diseases, including Parkinson's disease. General information on the drug development process is provided below.
Drug Development Process
Introduction
The process of developing, testing and gaining approval for new treatments involves many critical steps and typically can take ~10 years from preclinical development to drug approval. The following is a general description of the stages of drug development in the United States.
Preclinical Testing
Preclinical testing is conducted to evaluate the safety of an investigational treatment before administration to humans and to assess the treatment’s potential to impact a disease. In this stage, scientists test the treatment in laboratory and animal experiments to collect safety data and to evaluate biological activity against the targeted disease.
Clinical Trials
After preclinical testing is completed, the FDA reviews the available data and decides whether an investigational treatment can move forward into clinical trials in humans. If a treatment moves into clinical trials, there are three general stages of clinical research.
- Phase 1
In Phase 1 clinical trials, the investigational treatment is tested for the first time in humans. Phase 1 studies are most often conducted in healthy volunteers. The purpose of Phase 1 trials is to evaluate safety in humans at a range of doses. Phase 1 studies also provide pharmacokinetic (how the body affects the drug) and pharmacodynamic (how the drug affects the body) data.
- Phase 2
Phase 2 trials are conducted in patients and are designed to provide preliminary data on effectiveness and information on side effects. At this stage, different dose levels and regimens are often tested, which ultimately helps determine dosing for further trials.
- Phase 3
Phase 3 trials are designed to confirm treatment effectiveness and to monitor side effects. These trials are conducted in larger groups of patients and provide the information necessary to evaluate use of the treatment in the general patient population.
Approval & Post Marketing Studies
Once clinical trials are completed, a New Drug Application (NDA) is submitted to the FDA for review. The NDA contains all the scientific data that a company has gathered regarding the safety and effectiveness of an investigational treatment. The FDA reviews the NDA and if approved, the new treatment can be marketed and distributed.
Once the treatment is on the market, additional studies may be performed to evaluate treatment effectiveness and safety during routine use or to assess the treatment in new segments of the patient population.
External Resources
For additional information on drug development and the clinical research process, please refer to the following resources: