New medication also reduces by 40 percent the number of pulmonary exacerbations, the leading cause of death in cystic fibrosis patients
Results from two phase 3 clinical trials published today in the New England Journal of Medicine show that a new combination of medications can successfully treat the underlying cause of cystic fibrosis for patients age 12 and older with two copies of the F508del gene mutation – the most common form of the life-threatening, genetic disease found in over half of the cystic fibrosis (CF) population. Approximately 8,500 people in the U.S., and 22,000 people in North America, Europe and Australia, age 12 and older with cystic fibrosis carry this gene mutation.
The international trial, which studied more than 1,000 cystic fibrosis patients age 12 and older, revealed that a combination of the drugs Kalydeco (ivacaftor) and lumacaftor, an experimental drug that has not yet been approved by the Food and Drug Administration (FDA), successfully treated the defective CF protein and improved lung function. The drugs also helped patients achieve a 40 percent reduction in pulmonary exacerbations, the leading cause of death in cystic fibrosis patients.
“Being able to treat not just the symptoms, but the underlying cause of cystic fibrosis by targeting this specific gene mutation, is a major game changer because it is by far the most common in patients with this disease,” said Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and professor of Pediatrics at the University of Washington, who presented the study’s findings at the Center for Drug Evaluation and Research advisory committee meeting on May 12. “Previously, we found that Kalydeco treated just four percent of cystic fibrosis patients, but this new combination treats the defective CF protein for a much greater number of patients.”
The drugs, which work together to address the defective gene mutation by allowing the proteins within the cell to fold properly, reach the cell surface and then work as a salt channel, were given to patients over the course of 24 weeks in an effort to gain insight on how they may improve lung function and quality of life for people living with cystic fibrosis.
The trial included cystic fibrosis patients from Seattle Children’s and the University of Washington Medical Center. Today, the median predicted age of death for a person with the disease is in the mid-20s.
“What gives me tremendous confidence and excites me the most are the comprehensive health improvements seen in patients across all age groups, in all geographies and with all levels of disease severity,” said Ramsey, who was one of four lead authors on the trial and is a pioneer in cystic fibrosis treatment. “Patients can now have longer periods of good health, greatly improving their quality of life.”
The Latest on: Cystic Fibrosis
via Google News
The Latest on: Cystic Fibrosis
- Access to cystic fibrosis drug would save lives, says Nova Scotia womanon February 26, 2021 at 2:00 am
A Nova Scotia woman with cystic fibrosis says she has contemplated moving to Europe in a desperate attempt to gain access to a drug that is being hailed as a breakthrough.
- Cystic fibrosis: Life-saving Trikafta drugmaker announces it will submit Medsafe application, in talks with Pharmacon February 26, 2021 at 12:42 am
It's great news for young Kiwi Izaeah Twose, who's been relying lately on the kindness of strangers to stay alive.
- Translate Bio Announces Fourth Quarter and Full Year 2020 Financial Results and Highlights ...on February 25, 2021 at 1:31 pm
Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat or prevent debilitating or ...
- Translate Bio Announces Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Progresson February 25, 2021 at 1:21 pm
Additional interim data expected in early Q2 from Phase 1/2 clinical trial of MRT5005 for the treatment of cystic fibrosis (CF) -- -- Two infectious disease mRNA vaccine programs expected to enter ...
- Renowned Nutrition Expert Puts Rare Disease Day on the Radaron February 24, 2021 at 7:34 am
To raise awareness of Rare Disease Day on February 28, Envara Health founder and nutrition expert Virginia A. Stallings, M.D., highlights the nutritional challenges and therapies for people living ...
- Global Cystic Fibrosis Market Size 2021 Top Manufacturers, Share, Growth Factor, Latest Trends, Type and Application Forecast 2026on February 23, 2021 at 1:06 am
Global “Cystic Fibrosis Market” report is a fact-based market research and important information to companies, ...
- How do patients with cystic fibrosis respond to COVID-19?on February 22, 2021 at 8:13 am
Based on clinical data, patients with cystic fibrosis (CF) don't appear to be especially susceptible to COVID-19, and when they do get infected, they don't seem to get sicker. But Ruobing (Ruby) Wang, ...
- Registration open for 30-day fitness challenge benefiting those with cystic fibrosison February 20, 2021 at 11:45 am
For most of us, taking a deep breath of fresh air is one of the simple pleasures in life. Fresh air fills our lungs as we inhale and leaves our ...
- Cystic Fibrosis and COVID-19on February 16, 2021 at 1:31 pm
People with cystic fibrosis, or CF, don’t appear to be especially susceptible to COVID-19, and when they do get infected, they don’t seem to get sicker, based on clinical data so far. But Ruobing ...
- California boy with cystic fibrosis also battling coronavirus: ‘He doesn’t let anything get him down’on February 15, 2021 at 12:24 pm
A young California boy who was born with cystic fibrosis is also battling a COVID-19 infection, his mother said. Noah Schneider, 5, tested positive for the novel virus just ahead of the New Year, his ...
via Bing News