Patients suffering from Cushing's Syndrome can breathe a bit easily now. The US drug manufacturers Corcept Therapeutics has received orphan drug status for its Corlux from the Food and Drug Administration (F.D.A.).
Cushing's Syndrome is a disorder caused by prolonged exposure of the body's tissues to high levels of the hormone cortisol.
Symptoms vary, but most people have high blood sugar, high blood pressure, upper body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety and depression are common.
Cortisol is a normal hormone produced in the outer portion, or cortex, of the adrenal glands, located above each kidney. The normal function of cortisol is to help the body respond to stress and change. It mobilizes nutrients, modifies the body's response to inflammation, stimulates the liver to raise the blood sugar, and it helps control the amount of water in the body. Another adrenal cortex hormone, aldosterone, regulates salt and water levels which affects blood volume and blood pressure. Small amounts of androgens (male hormones) are also normally produced in the adrenal cortex. Cortisol production is regulated by adrenocorticotrophic hormone (ACTH), made in the pituitary gland, which is located just below the brain.
When too much cortisol is produced in the adrenal glands, or an excess is taken in treating other diseases, significant changes occur in all of the tissues and organs of the body. All of these effects together are called Cushing's Syndrome.
Sometimes called "hypercortisolism," it is relatively rare and most commonly affects adults aged 20 to 50. An estimated 10 to 15 of every one million people are affected each year.
It is precisely in treatment of such rare diseases the designation of orphan drug helps.
In the US an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ( or "orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States, prevailing in less than 5 per 10,000 in the community.
Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity on that drug for an extended time (seven years post-approval).
The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research. Under the act many drugs have been developed, including drugs to treat such diseases as multiple myeloma, bone marrow cancer.
Welcoming the FDA decision, Corcept said it was yet to finalise its strategies for Corlux but would start going about marketing the drug soon.