FDA approves cystic fibrosis drug Orkambi
SILVER SPRING, Md. — The Food and Drug Administration recently approved Vertex Pharmaceuticals’ Orkambi (lumacaftor 200 mg/ivacaftor), a new cystic fibrosis treatment that received designation from the FDA as a breakthrough therapy and as an orphan drug.
Orkambi is specifically for individuals with cystic fibrosis with a mutation (F508del) that disrupts water and chloride transportation in the body by producing an abnormal protein. The leading cause of the disease is when an individual inherits one copy of this mutation from each parent.
“The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis,” John Jenkins, director of the Office of New Drugs at the Center for Drug Evaluation and Research said. “Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis.”
The FDA approved Orkambi after a priority review, which is conducted in a shorter time than most reviews for drugs the agency believes can offer significant safety and efficacy improvements over currently available drugs.
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Report: PANTHERx expands specialty pharmacy business
PITTSBURGH — Specialty pharmacy PANTHERx has expanded its specialty pharmacy business and doubled its workforce, according to a local news report.
Founded in 2011, PANTHERx has expanded its pharmacological support services for organ transplant patients, the Pittsburgh Business Times reported, while adding medications for HIV, hepatitis C and skin conditions.
In less than a year, the company has also doubled its workforce to 40 full-time associates, according to the article.