Committed to Our Patients

At Connectyx, we are aspiring to grow into an organization that fulfills the unmet needs of patients with rare diseases through the development of pharmaceuticals and medical devices.

 

There are approximately 7,000 rare (also called orphan) diseases recognized in the United States. We are focusing on helping patients with these diseases who may have no or few treatment options.

 

Most patients know that pharmaceutical development can take a long time. In some cases, the FDA can fast-track an orphan drug, which could expedite the approval process. The roadmap for a new drug can be divided into three phases. In general, a new drug must pass through these phases successfully in succession.

Phase 1

    • 3 to 50 people
    • Typically healthy volunteers, but can also be people who have the disease
    • Evaluates safety and how the body breaks down the drug

Phase 2

    • 10 to 60 people
    • People who have the disease
    • Evaluates safety and effectiveness (the ability of a drug to produce a beneficial change)

Phase 3

    • 12 to 150 people
    • People who have the disease
    • Comparison of the long-term safety and effectiveness of the treatment of a drug to the current standard of care or a placebo

After a study is completed successfully, all information on a drug is provided to and reviewed by a government agency, like the United States Food and Drug Administration (FDA). If the research shows the drug is both safe and effective, it could be approved for use by prescription. As the Connectyx team endeavors to develop new treatment options, we will provide information on this website.

 

Rare Disease Organizations

 

If you or a loved one has a rare disease, here are a few resources you can explore:

 

https://rarediseases.org/

https://www.orpha.net/consor/cgi-bin/index.php