Orphan Medical Will Launch Sucraid For Sucrase Deficiency In June

Orphan Medical will launch Sucraid (sacrosidase) for treatment of the genetic disorder CSID in early June.

Sucraid is indicated "as oral replacement therapy of the genetically determined sucrase deficiency, which is part of congenital sucrase-isomaltase deficiency," labeling states. FDA approved Sucraid (NDA 20-772) April 9 as a "1P" new molecular entity priority application. The NDA was filed May 6 and reached "approvable" stage Nov. 6.

"Approximately 4%-10% of pediatric patients with chronic diarrhea of unknown origin have CSID," labeling states. Orphan Medical estimates the patient population at less than 1,000. The drug will have seven years of exclusivity under the Orphan Drug Act.

A price for Sucraid has not been announced, but other enzyme replacement therapies for orphan indications such as Genzyme's Ceredase and Enzon's Adagen cost over $100,000 per year. Orphan Medical has made arrangements with the National Organization of Rare Disorders to administer a patient assistance program for Sucraid.

CSID patients who lack adequate health insurance and pass a financial need test will be eligible to receive the drug free of charge through NORD.

CSID "is usually characterized by a complete or almost complete lack of endogenous sucrase activity, a very marked reduction in isomaltase activity, a moderate decrease in maltase activity and normal lactase levels," labeling states. As a result, "unhydrolyzed sucrose and starch are not absorbed from the intestine," which in turn can lead to loose stool, excessive gas, bloating, abdominal cramps, nausea and vomiting.

"Undiagnosed/untreated CSID patients often fail to thrive and fall behind in their expected growth and development curves," labeling adds.

"Previously, the treatment of CSID has required the continual use of a strict sucrose-free diet," labeling states. Some patients also avoid starch, Orphan Medical added. Sucraid does not replace the isomaltase and maltase starch-processing enzymes.

The approval was based on two double-blind crossover trials conducted in 54 patients, with only one of the studies demonstrating clear clinical efficacy.

In a 28-patient trial, sacrosidase patients experienced "significantly fewer total stools and...lower total symptom scores, the primary efficacy endpoints," labeling states. "In CSID patients up to three years of age, 86% became asymptomatic. In patients over three years of age, 77% became asymptomatic."

The second trial, which used Sucraid concentrations significantly lower than the therapeutic dose of 8,500 International Units, had "inconsistent results with regards to the primary efficacy parameters," labeling states. However, patients receiving Sucraid in both trials had "a marked decrease in breath hydrogen output" compared to placebo.

Adverse reactions to Sucraid include abdominal pain, vomiting, nausea, diarrhea, constipation, insomnia, headache, nervousness, and dehydration. One four-year old boy who was asthmatic experienced "severe wheezing, 90 minutes after a second dose of sacrosidase" and was admitted to the ICU.

For this reason, labeling cautions that initial doses of Sucraid should be administered "near a facility where acute hypersensitivity reactions can be adequately treated." Another option is to test the patient "for hypersensitivity to Sucraid through skin abrasion testing," labeling advises.

The drug is contraindicated in patients "known to be hypersensitive to yeast, yeast products, or glycerin (glycerol)," labeling states, because its active ingredient, sacrosidase, is derived from yeast.

Labeling warns that "Sucraid should not be reconstituted or consumed with fruit juice, since its acidity may reduce the enzyme activity." Sucraid bottles should be discarded "four weeks after first opening due to the potential for bacterial growth."

Sucraid will be available in 118 mL plastic bottles, with each mL of solution containing 8,500 IU of sacrosidase. The recommended dosage is 1 mL per meal or snack for patients up to 15 kg and 2 mL for patients over 15 kg.

Orphan Medical licenses Sucraid from Hartford Hospital in Connecticut. The product is manufactured for Orphan by Gloucester, Mass.-based NutraMax Products. Minneapolis-based Orphan will market the product directly to pediatric gastroenterologists.