This Experimental Drug Just Received Orphan Drug Designation For Congenital Adrenal Hyperplasia

According to a story from, the experimental treatment SPR001 was just granted orphan drug status by the Food and Drug Administration (FDA). The new treatment is currently being developed by Spruce Biosciences, a pharmaceutical company. The drug was granted the status for the treatment of a rare condition called congenital adrenal hyperplasia.
Congenital adrenal hyperplasia (CAH) are several different diseases caused by mutations in genes responsible for the production of a variety of substances from the adrenal gland. The disease often involved excessive or insufficient of sex steroid which can alter the primary and secondary sex characteristics in those that are affected. The symptoms can range widely depending on the type of CAH and the patient’s sex. The types of disease are classified by which enzyme is deficient. Symptoms include ambiguous genitalia (it is difficult to identify the genitalia the person has, more common in females), rapid growth and the early development of public hair, delayed puberty or failure of it to occur, and infertility. To learn more about these diseases, click here.

Orphan drug designation is a valuable step in the development process for SPR001. The designation was first developed as a way to provide additional incentive for pharmaceutical companies to develop drug treatments for rare or unusual diseases. Normally, companies have only minimal incentive because a treatment will be in low demand by default if the disease it is meant for is considered rare. The orphan drug designation gives incentive by exempting the company from certain taxes and fees and also gives the exclusive rights to continue with the drug development. The designation is typically reserved for drugs that show exceptional promise and will treat a disease that had few or no currently available treatment options.

Current treatment options vary depending the form of CAH. It typically involves supplementing the production of the enzyme that this patient is lacking or supplying a treatment that will counteract the excessive production of enzymes that is present in other types. There is currently no available FDA approved therapy that can cure or reverse the cause of the disease, so the need for a new treatment is urgent.

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