Rifaximin Announced as Orphan Drug May Assist in Treating Sickle Cell Disease
Posted On December 11, 2020
The FDA or the U.S. Food and Drug Administration has designed an oral antibiotic called Rifaximin, which is an orphan drug known to be a potential treatment to ease pain crisis due to SCD or sickle cell disease, as stated by a press release. The gastroenterology arm of Bausch Health Companies, Salix Pharmaceuticals, noted that a phase 2 trial to test Rifaximin in SCD patients is expected to take place next year, in a new formulation of the disorder.
Therapies having the potential to improve medical health care for rare disorders are awarded the orphan designation. It supplies incentives to the organizations for further advancements of the treatment, including seven years of marketing exclusivity, trail protocol assistance, and fee waives upon approval.
The symptoms of sickle cell disease include clogging of small blood vessels and periods of extreme pain due to the clumping together of sickle-shaped RBCs. These episodes referred to as VOC or vaso-occlusive crisis are known to be common and the primary reason behind hospital admission of people having SCD.
Aged and activated neutrophils, which are a type of immune cells, partake in these crises. According to various studies, heightened amounts of these cells are mostly related to abnormal gut microbiota and intestinal damage in sickle cell disease patients. Several functions in the body, such as inflammatory processes are regulated, by the collection of bacteria present in the gut. Changes in the amount and type of bacteria in the gut microbiota may result in the activation of damaging immune cells by significantly impacting these processes.
Rifaximin is the antibiotic preferably used in the treatment of traveler’s diarrhea and numerous other intestinal diseases. The severity and frequency of VOCs are lowered, with the shifting of intestinal bacteria towards the ‘good’ bacteria, which lessens the neutrophil activation in the medication. The potentiality of rifaximin to reduce the frequency of hospitalized cases due to painful VOCs in sickle cell disease patients was investigated by a Phase 2 clinical trial that took place in 2018.
According to the research results, the use of rifaximin significantly reduced inflammatory markers and eased intestinal injury, including the number of neutrophils.
Rifaximin drug can be used as an essential medicine to reduce pain in sickle cell patients; thus, has the potential to create a positive impact on the global sickle cell disease treatment market.
Global Sickle Cell Disease Treatment Market Report