Vertex Pharmaceuticals announced the FDA has approved ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population. ORKAMBI oral granules are available in two dosage strengths (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. ORKAMBI oral granules should be available for fulfillment within 2 to 4 weeks.

“For the first time, children ages 2 through 5 who have the most common form of CF have a treatment for the underlying cause of their disease,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We believe it is important to treat the underlying cause of the disease as early as possible and this approval is another significant milestone in our journey to bring effective medicines to all people living with CF.”

This FDA approval is based on a Phase 3 open-label safety study in 60 patients that showed treatment with ORKAMBI was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24 (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6, n=49). Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study. The most common adverse event (≥30%) was cough (63%); most adverse events were mild or moderate in severity.

“Cystic fibrosis is a systemic, multi-organ, progressive disease that is present from birth,” said Dr. John McNamara, Medical Director of the cystic fibrosis program at Children’s Minnesota hospital and lead study researcher. “Research suggests ORKAMBI could impact CF outcomes in patients as young as two years old. This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before.”

ORKAMBI was already approved in the U.S. for the treatment of CF in patients ages 6 and older who have two copies of the F508del-CFTR mutation. A Marketing Authorization Application (MAA) line extension for ORKAMBI in children ages 2 through 5 years has been submitted to the European Medicines Agency (EMA) with a decision anticipated in the first half of 2019.

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

VRTX stock chart

We are 11% on VRTX since I profiled this stock on February 7, 2018 in an article entitled Vertex Pharmaceuticals PDUFA February 28 2018.

Vertex Pharmaceuticals released its quarterly earnings on July 25, 2018. The pharmaceutical company reported EPS of $0.94 crushing the $0.40 estimate. Revenue also crushed estimates coming in at $752.20 million for the quarter versus the $680.86 million estimate. Vertex Pharmaceuticals’ revenue was up 38.2% year-over-year.

Looking at the last year, revenue has grown by 23%.

Revenue has been growing at an excellent clip of 16.70% on average over the past 5 years.

I continue to like VRTX stock for a long-term buy and hold.

Disclosure: As Premium members know, I am long VRTX stock.