FDA Approves Two New Orphan Drugs: Rubraca and Spinraza

Roni Shye
Roni Shye, PharmD BCGP BCACP, is a licensed pharmacist in the states of Florida, Ohio, and Pennsylvania.
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Recently, the FDA approved two new orphan drugs, Rubraca and Spinraza.

But first, what is an orphan drug?

An Orphan drug is a medication used to treat a rare disease. By definition, a rare disease is one that affects less than 200,000 patients in the United States, according to the Orphan Drug Act or 1983.

Because some diseases are so rare, a lot of pharmaceutical manufacturers are hesitant to develop medications for the small amount of people that are affected. However, the FDA provides financial incentives to pharmaceutical companies willing to research and develop medications for rare disease.

Some rare diseases that are typically treated with orphan drugs include blood disorders, autoimmune disorders, generic disorders, and spinal muscular atrophy.

What is Rubraca indicated for?

Rubraca is for the treatment of patients with a certain type of ovarian cancer. It has been approved for women whose tumors have the BRCA gene mutation.

What is Spinraza indicated for?

Spinraza is the first and only treatment for spinal muscular atrophy in children in adults.

Spina muscular atrophy is a genetic disease that affects the part of the nervous system that controls voluntary muscle movement. Spinal muscular atrophy causes weakness of muscles used for activities like crawling, walking, sitting up, and controlling head movement. In severe cases, the muscles used for breathing and swallowing are affected.

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