ICYMI: Cystic Fibrosis Drug TRIKAFTA Accepted for Priority Review in Canada for Patients Aged 6-11

 

In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late October 2021, Vertex Pharmaceuticals Incorporated (“Vertex”), the drug’s developer, submitted a supplement to its New Drug Submission. This supplement was designed to approve TRIKAFTA use for patients aged 6-11. The news release explains that Health Canada accepted the supplement for Priority Review, which offers a shortened review process of 180 days.

TRIKAFTA

So what exactly is TRIKAFTA? According to the Cystic Fibrosis Foundation (CFF), TRIKAFTA is:

a “triple-combination therapy” consisting of three different modulators — tezacaftor/ivacaftor (which make up Symdeko®) combined with elexacaftor. Modulators work by helping to fix defective CFTR protein.

The CFF also explains that TRIKAFTA has shown a promise in increasing lung function for those with cystic fibrosis.

Cystic Fibrosis (CF)

Caused by CFTR gene mutations, cystic fibrosis (CF) is a rare and progressive genetic disorder which can affect multiple systems throughout the body, including the lungs, liver, and gastrointestinal tract. Because it is inherited in an autosomal recessive pattern, patients must inherit one defective gene from each parent.

Normally, our bodies produce “healthy” mucus, which is slippery. CFTR gene mutations affect the CFTR protein, which regulates salt movement. Because of this, those with CF have thick, sticky mucus which accumulates in their body. This thick mucus can prevent the release of digestive enzymes, prevent nutrient absorption, and cause frequent infections and breathing difficulties. Risk factors include being Caucasian and having a family history of CF.

Symptoms associated with cystic fibrosis include, but are not limited to:

  • Salty-tasting skin
  • Exercise intolerance
  • Recurring sinusitis
  • A persistent cough which produces mucus
  • Wheezing
  • Shortness of breath and/or difficulty breathing
  • Poor weight gain
  • Greasy and foul-smelling stools
  • Frequent or repeated lung infections
  • Constipation
  • Intestinal blockages
  • Infertility (in males)

Some complications of cystic fibrosis include:

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email