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Vertex Submits Cystic Fibrosis Drug To FDA For Testing

By Julie M. Donnelly, Boston Business Journal
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BOSTON (CBS) – Vertex Pharmaceuticals Inc. has filed for U.S. Food and Drug Administration approval for its potential therapy for cystic fibrosis, and has asked for an accelerated review of the drug candidate.

The Cambridge, Mass-based company announced that if approved, the drug would be called Kalydeco.

If the FDA grants priority review to the potential treatment, it could be approved in as little as six months.

If successful, Kalydeco would be the second wholly owned drug on the market for Vertex.

Its drug for hepatitis C, Incivek, was approved in May.

Vertex also plans to submit Kalydeco to European regulators by the end of October.

“These regulatory applications are a reflection Vertex’s 13-year research and development effort and the commitment of hundreds of doctors, nurses, patients and their caregivers who participated in the studies of Kalydeco,” Vertex Chief Scientific Officer Peter Mueller said in a statement.

“We look forward to working with U.S. and European regulatory agencies to make Kalydeco available as quickly as possible.”

Kalydeco is unique in that it targets the defective protein that causes cystic fibrosis, rather than the symptoms of the disease, as currently approved treatments do.

It is also a personalized medicine, targeting the 4 percent of CF patients who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

There are about 1,200 people in the U.S. with this mutation.

Phase 3 studies on the drug candidate showed that it had “significant and sustained improvements in lung function and other measures of disease” for the patient group with the mutation, according to the company.

Lisa van der Pool of the Boston Business Journal reports

Fewer people in the Kalydeco treatment groups than in the placebo groups discontinued treatment due to adverse events, the company said, and most side effects were mild to moderate.

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