This FDA Nod Is a Game Changer for Patients: Could It Equal Billions for Vertex Pharmaceuticals?

Vertex Pharmaceuticals (NASDAQ: VRTX) is already a rock star in the world of cystic fibrosis (CF) treatment. The company's drugs have been life-changing for children and adults living with the disease. And that's resulted in billion-dollar revenue and profit for Vertex. It's also helped the company build up more than $11 billion in cash to support development of new CF drugs and candidates in other areas.

But one thing has been missing from this bright picture. And that's treatment for the very youngest of patients: babies from one month through three months old. Until now. The U.S. Food and Drug Administration (FDA) recently approved Vertex's Kalydeco for use in this age group. It's a clear game changer for patients. Could it also equal billions for Vertex? 

It's important to first look at why Vertex's CF drugs are so successful. In CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene lead to the production of a faulty protein. This protein can't do its job right -- and that causes CF's devastating symptoms. Vertex's CFTR modulators help the protein function properly. So, these products truly are extending life and quality of life.

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Source Fool.com