FDA Approves Procysbi to Treat Genetic Condition

The U.S. Food and Drug Administration has given a nod to Novoto's 'Procysbi' for treating nephropathic cystinosis in kids and adults. The drug was given the 'orphan product' designation as it was meant to treat a rare disease or condition.

Apart from Procysbi, the FDA-approved drugs to treat this rare genetic condition include Cystagon which was approved in 1994, and Cystaran eye drops, approved in 2012.

A rare genetic disorder, cystinosis affects nearly 500 patients in the U.S. It causes a protein building block known as cystine to build up in every cell of the body. The presence of cystine causes kidney problems, making the body lose excess sugar, salt and proteins every time the person urinates.

Procysbi is a delayed-release drug that is used to treat patients who are 6 years or older. It is meant to be taken every six hours in order to control the levels of cystine.

"Procysbi is the only delayed-release product approved by FDA to treat nephropathic cystinosis, offering patients with this rare disease an important new treatment option," Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products, Center for Drug Evaluation and Research, FDA, said in a press statement.

To test the safety of the drug, a study was conducted on 43 adults and pediatric patients who were victims of nephropathic cystinosis. Patients received either Cystagon or Procysbi for nearly three weeks before they were started on another product for the next three weeks.

On analyzing the results of a blood test, researchers noticed that Procysbi was as effective as Cystagon in controlling cystine levels.

The most common side effects of the drug were nausea, bad breath, abdominal pain, upset stomach, dizziness, drowsiness and headache. Some of the serious side effects were ulcers or bleeding of the stomach or intestine, altered mental state, seizures, skin rashes and allergic reactions, reports FDA.