Short bowel syndrome is a malabsorption disorder that causes the body to not get the fluids or nutrients it needs. It can cause dehydration and malnutrition, in addition to other complications.
Gattex is a drug approved to treat adult patients with SBS who require parenteral support. Parenteral support means the patient has to receive the nutrients they need intravenously. Unfortunately, there have been little to no options for pediatric patients who require IV support.
Hopefully, that will change soon.
Gattex was sent in for FDA approval for pediatric patients in September of this year and we’ve just received word that its application has been accepted for consideration. We should know by March of 2019 whether or not children will get access to Gattex.
Specifically, approval would mean patients aged 1 to 17 would be able to receive the injection.
The primary goal of this new development is, of course, to provide pediatric patients with the nutrition they are lacking. But, it could also potentially help to foster independence. Many individuals living with SBS are reliant on parenteral care. If this treatment is approved, it may help give patients greater control of their own condition and improve their overall well-being.
Efficacy of the Drug
Gattex’s application is supported by two phase 3 trials that showed the drugs safety and efficacy.
One of the studies found that over 50% of patients treated with the drug achieved a reduction in parenteral support of 20% or more within the first 24 weeks. For those who did not receive the drug, only 11% had this rate of improvement. Even more incredibly, 5 children in that study were completely weened off of parenteral support at the end of 22 weeks. In the placebo group, no children had that success.
Approval of this drug would not only mean a better treatment option for pediatric patients, it could greatly improve their quality of life.
You can read more about this drug and the studies that went into its FDA application here!
Keep your fingers crossed for its approval and check back in March to hear the news!