FDA Orphan Drug Designation

Posted by Michael Gralinski, Chief Executive Officer at CorDynamics on January 30th, 2012

There are currently about 7,000 orphans out there waiting to be adopted, to be supported, to be cured. Many of us don’t even know their names: Kawasaki. Porphyria. Pulmonary Arterial Disease.

Orphan Status is a designation given by the FDA to identify rare diseases with unmet medical needs but with potentially promising compounds currently being researched. Orphan drugs receive expedited FDA reviews and fast tracks to clinical trials, providing hope for cures, treatments or comfort.

>>>>>  Nearly 2,400 experimental compounds have orphan drug designations.

>>>>>  So far, only 367 of these orphan drugs have been approved by the FDA.

Experts agree, coordinated collaboration and resources on all fronts will be the key to finding a potential drug therapy for these orphan drugs. Collaboration is also essential for the rare diseases still searching for enough answers to even begin the orphan drug development stage.

You may ask, what can I do to help? What can my organization do?

Adopt An Orphan

  • Pick an orphan disease with a compound in the works or a rare disease to adopt.
  • Show your support and learn more about the disease, follow related organizations on Facebook or in the news.  One that we follow on Facebook is Two Hearts Rock and Global Genes Project.
  • Donate your time, participate in a sponsored event, or fundraiser.
  • “Wear That You Care” on World Rare Disease Day, February 29th. Wear “jeans” for “genes” and encourage your workplace, schools, and sport teams to do so too to raise awareness for the Global Genes Project.

At CorDynamics, we sponsored a fundraising event for Hope4Bridget and Batten Disease (a rare disease) as well as continue to work with our clients on promising compounds for the potential treatment of Pulmonary Arterial Hypertension (an orphan designated rare disease).

Working together, we will continue to adopt more of these orphan diseases and meet more unmet medical needs. To learn more about this issue and how you can help, jump into today’s blog hop sponsored by the R.A.R.E Project.


4 Responses to “FDA Orphan Drug Designation”

  1. January 30, 2012 at 6:40 pm, mindy said:

    Thank you for this post! We have benefited so much from Orphan Drug status, which has led to medications that work effectively control our son’s disorder. I don’t know what we would have done without the tireless work of others that spread awareness and work so hard to find treatments for rare disease.

    Pick an orphan..I like that.

    Mindy~(a rare disease mama)


    • January 30, 2012 at 7:11 pm, CorDynamics said:

      Hello Mindy,

      Glad you liked the post. Stories like yours inspire our team to continue to support drug development research with our science and those suffering from rare diseases with our hearts and sponsorship.

      Take care and best regards!


  2. February 03, 2012 at 1:40 am, Vicki said:

    Thank you for your post. How do we find out which rare diseases have compounds in the works? Thank you for the job that you do.


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