On November 27, biotechnology company Vertex Pharmaceuticals Inc. (“Vertex”) announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The European Commission (EC) approved the use of this therapeutic combination for patients ages 6 and up. Specifically, SYMKEVI and KALYDECO are designed for patients with at least one F508del gene mutation. Overall, this is one of the more common genetic mutations associated with CF.
SYMKEVI and KALYDECO
Together, SYMKEVI and KALYDECO represent one of the first therapeutic options for CF that addresses this specific genetic component. Although it is already approved for patients over the age of 12, this approval marks a new available treatment for younger patients. The combination will first be available to patients in Germany. However, it will later be available in Denmark, Ireland, and the United Kingdom. Outside of these countries, Vertex is working to bring SYMKEVI and KALYDECO to patients in need.
The drugs work by normalizing defective CFTR protein, enabling it to reach cells, and prompting it to function correctly.
Cystic Fibrosis (CF)
As a recessive genetic disorder, cystic fibrosis is caused when someone inherits two defective or mutated CFTR genes, one from each parent. There are a multitude of gene mutations that can impact salt regulation, leading to cystic fibrosis. Normally, our systems have “healthy” mucus, which is slippery. But people with cystic fibrosis have thick, sticky mucus that accumulates in the system. As this mucus accumulates, it clogs airways, traps bacteria, and causing a multitude of respiratory issues. Additionally, it stops digestive enzymes from being released, causing poor nutrient absorption. Cystic fibrosis affects Caucasians at a higher rate than other ethnicities. Symptoms include:
- Male infertility
- Frequent lung infections
- Chronic coughing or wheezing
- Shortness of breath / difficulty breathing
- Poor growth
- Abdominal pain
- Difficulty gaining weight
- Exercise intolerance
- Stuffy nose
- Salty skin
- Constipation or greasy-looking bowel movements