Synergy Pharmaceuticals Obtains $5.64M Financing

You might want to take a friend or family member with you to the appointment to help you remember information. Before your appointment, make a list of: Symptoms and when they started. Include anything that makes symptoms worse or better. All medicines, vitamins, herbs and supplements that you or your child take. Include the doses. Family history, such as whether anyone in your family has cystic fibrosis. Treatment you or your child have had for CF, if any. Include what the treatment was and if it helped. Any other medical conditions and their treatments. Questions to ask your healthcare professional. Questions to ask may include: What is likely causing these symptoms? What kinds of tests are needed? What treatment do you recommend? I or my child have other health conditions. How will cystic fibrosis affect them? Are there any limits needed? Feel free to ask other questions during your appointment.

Cystic fibrosis (CF) is a genetic disorder, which means you get it from your parents at birth. It affects your lungs, pancreas, and other organs. CF changes the way chloride (salt) moves through the cells of your body. This causes the mucus (which should be thin and slippery) in various organs to become thick and sticky. Over time, this thick mucus builds up inside your airways, making it hard to breathe. The mucus traps germs and leads to infections and inflammation. It can also cause severe, long-term damage to the lungs and lead to respiratory failure (inability to breathe normally) and death. In the pancreas, the thick mucus caused by CF prevents the release of digestive enzymes when you eat. This leads to malnutrition and poor growth. CF can also cause liver disease, reproductive problems, and cystic fibrosis-related diabetes (CFRD). More than 40,000 people in the U.S. live with CF. Doctors diagnose about 1,000 new cases each year. Today, more than half of the CF population is aged 18 or older, and new treatments have expanded the life expectancy by decades.

Learn more from pulmonologist Sarah Chalmers, M.D. Cystic fibrosis (CF) is a condition passed down in families that causes damage to the lungs, digestive system and other organs in the body. CF affects the cells that make mucus, sweat and digestive juices. These fluids, also called secretions, are usually thin and slippery to protect the body's internal tubes and ducts and make them smooth pathways. But in people with CF, a changed gene causes the secretions to become sticky and thick. The secretions plug up pathways, especially in the lungs and pancreas. CF gets worse over time and needs daily care, but people with CF usually can attend school and work. They often have a better quality of life than people with CF had in past decades. Better screening and treatments mean that people with CF now may live into their mid- to late 50s or longer, and some are being diagnosed later in life.

A sweat test is used to diagnose cystic fibrosis (CF).

The effectiveness and safety of Alyftrek for cystic fibrosis was studied in two randomized trials (Trials VX20-121-102 and VX20-121-103). These studies compared Alyftrek to another standard treatment for cystic fibrosis ( elexacaftor /tezacaftor/ivacaftor). People in the studies first received elexacaftor/tezacaftor/ivacaftor for four weeks, then were assigned to continue this treatment or switch to Alyftrek for 52 weeks. Trial VX20-121-102 included 398 people with F508del-minimal function genotypes. The median (middle) age was 31 and 41% of people were female; most people in the study were White (97%), while 1% were Black or African American, and <1% were Asian. Trial VX20-121-103 included 573 people with F508del-F508del, F508del-residual function, F508del-gating, or other responsive mutations. The median age was 33.1 and 49% of people in the study were female. Most people were White (93%), 1% were Black or African American, and <1% each were Southeast Asian, other Asian, or American Indian/Alaska Native. The study found that Alyftrek was similar to elexacaftor/tezacaftor/ivacaftor in improving lung function (measured by forced expiratory volume in 1 second [FEV1 %]), with similar results seen in both studies. Additionally, Alyftrek led to greater reductions in sweat chloride, a key measure of CFTR function, in both studies. The results of these studies suggest that Alyftrek is a promising alternative to elexacaftor/tezacaftor/ivacaftor, offering similar lung function benefits and helping CFTR to work better. The safety profile of Alyftrek was similar to that of elexacaftor/tezacaftor/ ivacaftor . The most common side effects included worsening lung infections (28%), cough (23%), COVID-19 (22%), and the common cold (nasopharyngitis; 21%). Serious side effects were similar between groups, with 14% of people treated with Alyftrek and 16% of people treated with standard treatment experiencing severe reactions. High liver enzymes were slightly more common in the Alyftrek group, but overall safety findings were similar to those seen in other studies. Your results may differ from what was seen in clinical studies.