Adopting Orphan Diseases and Drugs
What Does it Mean to Drug Developers?
Orphan Status is a designation given by the FDA to identify diseases with unmet medical needs and promising compounds in these areas with the potential of providing cures, treatments and comfort.
From a business standpoint, the numbers shouldn’t be ignored. From the patient perspective, the need is paramount.
- More than 7,000 diseases have orphan status.
- An estimated 25 million Americans are afflicted with an orphan disease.
- Nearly 2,400 experimental compounds have orphan designations.
- So far, only 367 of these drugs have been approved.
For those of us involved in drug development, an orphan drug designation is a game changer and for patients it’s a cause for hope. Orphan Drugs receive expedited FDA reviews and fast tracks to clinical trials. It’s a process that’s been in place since the 80′s and has been used often in cancer trials. With an emphasis on collaboration, researchers are pooling resources and findings on other types of rare diseases as well.
At CorDynamics, we’ve done extensive preclinical and discovery studies for clients researching treatments for pulmonary arterial hypertension—an orphan disease. PAH is characterized by increased blood pressure in the arteries of lungs, causing dizziness, shortness of breath and can lead to heart failure.
On the charitable front, CorDynamics is sponsoring an event this month in the hopes of raising funds for Batten Disease— another devastating orphan condition.
Working together, we continue to strive to help our clients adopt more of these orphan diseases and meet more unmet medical needs.
Regards,
Dr. Michael Gralinski, CorDynamics CEO
Related Posts via Categories
- Tags:
- Batten Disease,
- FDA,
- Orphan Drugs,
- PAH,
- Pulmonary Arterial Hypertension Drug Discovery,
- Rare Disease
