Scientists are studying 2 main types of therapies:

CNP Analogues

CNP (which stands for C-type natriuretic peptide) is a hormone that plays an important role in bone growth.

CNP activates the NPRB receptors, (natriuretic peptide receptor B), reducing the effects of the overactive FGFR3 receptors, which tell the bones to stop growing.

Scientists are conducting clinical trials on therapies that are attempting to give the body more CNP to see if the overactive FGFR3 receptors can be countered, allowing bones to grow and develop. In these studies, scientists are also looking at what impact more CNP has on how bones grow and develop as well as what the risks may be.

The safety and effectiveness of these therapies being studied are not known.

The Decoy Method

In clinical trials, scientists are studying the “decoy method” for achondroplasia.

Decoys are designed to block the molecules that normally activate the FGFR3 receptors.

Clinical trials are being conducted to help scientists attempt to determine what happens if the molecules can’t reach the receptors. Scientists are looking at the impact that could have on bone growth and development, along with what the risks may be.

The safety and effectiveness of these therapies being studied are not known.

Research is being done to see if these therapies:

  1. Can reduce the signals from FGFR3 receptors (fibroblast growth factor receptor 3), which slow bone growth
  1. Are safe and effective in people with achondroplasia

What are clinical trials?

Scientific advancements in medicine come to life through clinical trials. They are key to the research and development of any new therapy. The goal of a clinical trial is to evaluate whether the therapy being studied is safe and effective.

Clinical trials take place in steps, or phases.

Depending on what is being measured, each step can last for many years or even decades. Scientists often make choices about what to measure so the results can be seen in a shorter span of time.

  • Phase 1

    A therapy is studied in a small group of usually healthy volunteers for several months to test its safety and to learn about any side effects.

  • Phase 2

    The therapy is studied in a group of people who are affected by the condition to look at its safety and whether it works. This phase also determines what dose of the drug should be taken into the next phase of research to study safety and effectiveness.

  • Phase 3

    This is the “pivotal trial.” A phase 3 study tests the therapy in people with the condition by comparing it either against a placebo (no therapy) or another standard treatment. This is usually done in a randomized, blinded way. This means that neither the patient nor the physician knows whether a study participant is on the new therapy or not. The therapy is studied over a longer period of time—usually several years—during this phase.

  • FDA/EMA approval

    The US Food & Drug Administration (FDA) or the European Medicines Agency (EMA) evaluate the information from the 3 clinical trial phases, reviewing the risks and efficacy, to determine if the therapy is approved for use by the public.

  • Phase 4

    If the therapy gets approved, researchers can conduct phase 4 studies to gather more information and track how safe the therapy is for the people using it.