According to a story from BioPortfolio, the biotechnology company Vertex Pharamceuticals, Inc. recently announced that the long term use of its innovative cystic fibrosis treatment Orkambi will be covered in Sweden; this reimbursement will go into effect on July 1st. Orkambi is approved for treating cystic fibrosis patients aged six or older. The success of the agreement could provide a workable model for similar arrangements to be made in other countries as well.
Cystic fibrosis is a genetic disorder that can cause wide ranging systemic effects, but it is mostly characterized by the build-up of abnormally thick, sticky mucus in the lungs and airway. This situation makes the patient far more vulnerable to lung infection compared to a healthy person. Cystic fibrosis is caused by a mutation that causes normally thin secretions to thicken instead. Symptoms include coughing mucus, breathing problems, infertility in men, sinus and lung infections, fatty stool, clubbed digits, and poor growth. The severity and degree of symptoms varies from patient to patient. Most people are on antibiotics for much of their lives, and there are other medications and procedures that can help maintain lung function. A lung transplant may be an option for some. Orkambi is the only drug that treats the underlying cause. Most patients survive to around age 40. To learn more about cystic fibrosis, click here.
While Orkambi is an important step forward in cystic fibrosis treatment, it really will only help those patients that have two copies of a mutation called F508del. The drug was first approved by the FDA in 2015. The drug is a combination of two different active components that can cause a slight improvement in lung function and can also reduce the need for hospitalization. This is an orally administered treatment, ensuring that quality of life for patients while using the drug is not a major obstacle. Orkambi has attracted some controversy because of its high price, which has stalled the approval process in some places.