Orphan Drug Designation Granted to Therachon’s Drug Apraglutide by FDA for Treatment of Short Bowel Syndrome

A recent article in CheckOrphan reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the synthetic GLP-2 analog apraglutide for the treatment of short bowel syndrome (SBS). Once a therapy has achieved Orphan Drug Designation it qualifies for incentives that will enable it to advance clinical development.

About Short Bowel Syndrome

Short bowel syndrome (SBS), a severe chronic condition affecting between 20,000 to 40,000 patients in the U.S. and Europe, is characterized by the complete loss of intestinal function which can be life- threatening. Malnutrition and malabsorption are major factors.

It is believed that SBS is the result of congenital abnormalities, major intestinal resection due to chronic inflammatory bowel disease, or from other acute events.

SBS patients usually require between 10 to 15 hours of intravenous drip feeding per day. This type of parenteral feeding is often associated with blood clots and infections.

About Apraglutide (FE 203799)

Apraglutide, a synthetic GLP-2 analog representing the newest technology in its field, tested successfully in a Phase 1 Single Ascending Dose and Multiple Ascending Dose clinical trial in healthy volunteers. The goal of administering an ascending dose is to determine the safety and maximum tolerated dose.

Apraglutide has been proven to be superior in the area of pharmacokinetics such as absorption distribution, metabolism, and excretion.  It is also easy to use with a once-a-week dosing regimen.  The drug is now being studied in two Phase 2 trials for short bowel syndrome.

About Therachon

Therachon’s mission is to develop best-in-class therapies and to treat the root causes of rare diseases that have unmet medical needs.

Upon acquiring GLyPharma Therapeutic Inc. in 2018, Therachon, a clinical-stage biotechnology company, entered the realm of multi-asset rare diseases with the addition of apraglutide.

The company is committed to continuing its programs in rare conditions that exhibit biological root causes in short bowel syndrome and achondroplasia, which is the most common cause of dwarfism.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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