FDA Approves Sacrosidase to Treat Congenital Sucrase-Isomaltase Deficiency

Sacrosidase was approved by the FDA to treat congenital sucrase-isomaltase deficiency, a condition that inhibits people from breaking down sucrose.

The FDA approved sacrosidase (Sucraid; QOL Medical, LLC) single-use containers to treat genetically determined sucrase deficiency, which is a part of congenital sucrase-isomaltase deficiency (CSID), in patients who weigh more than 33 pounds (15 kg). With this FDA approval, sacrosidase is available in both multi-dose bottles and single-use containers.1

"FDA's approval of Sucraid single-use containers, with 3-day room temperature stability, offer greater convenience for CSID patients on the go," said Tao in a press release.1

As the only pharmaceutical treatment of CSID in children and adults with genetically determined sucrase deficiency, sacrosidase is an oral solution and enzyme replacement therapy that relieves gastrointestinal symptoms which occur because the intestines cannot breakdown or absorb sucrose (table sugar), which is a common additive to many processed foods.1

"If untreated, patients living with this chronic condition suffer from debilitating symptoms. We are always looking for ways to improve their quality of life. These new single-use containers will make it easier for them to manage their medication," said Weng Tao, MD, PhD, COO of QOL Medical, LLC, in the press release.1

Symptoms of CSID can overlap with symptoms of IBS, which includes chronic abdominal pain, gas, loose stool, and diarrhea. However, CSID has distinct symptoms of explosive watery diarrhea, failure to thrive, diaper rash, irritability, and acidic stools.1

Humans naturally produce an enzyme called sucrase to break down sucrose in the small intestines, as sucrose is too large to be broken down by itself. Sucrase breaks sucrose into 2 smaller sugar molecules called glucose and fructose, which are more easily digested and absorbed into the blood to be used as energy.2

The sucrase-isomaltase enzyme is one of multiple enzymes that break down sucrose and starches—which are larger compounds than sucrose. Individuals with CSID are usually missing (or have a poor functioning) sucrase-isomaltase enzyme.2

If someone is unable to properly break down or absorb foods with sucrose (and sometimes starches) in the small intestines, the stomach will not empty food into the small intestines as quickly. This can inhibit the digestion of nutrients from starches, sugars, fats, and proteins, which can lead to issues with hormone regulation, low body weight, malnutrition, and GI symptoms.2

Additionally, GI symptoms can also manifest from gut bacteria, as the unabsorbed sucrose may feed bacteria, or microflora, who live in the large intestine. When bacteria help to break down sucrose without the enzyme sucrase, the bacteria use fermentation to accomplish this. The byproducts of fermentation are gases, organic acids, and molecules which can lead to swelling, abdominal pain, and water retention in the intestines, which can produce watery diarrhea.2

References

  1. QOL Medical, LLC,. QOL Medical, LLC receives FDA approval of Sucraid® (sacrosidase) Oral Solution single-use containers for patients with Congenital Sucrase-Isomaltase Deficiency (CSID). PR Newswire. September 6, 2022. Accessed on September 7, 2022.
  2. Sucraid (sacrosidase) Oral Solution. Sucraid website. Accessed on September 7, 2022. https://www.sucraid.com/about-csid/