The orphan drug designation is given to investigational therapies that have the potential to treat conditions that affect no more than five out of every 10,000 people in the EU. This designation makes Seelos eligible for certain incentives, including assistance designing protocols for studies, reduced regulatory fees, and market exclusivity if the therapy is approved.
Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is caused by mutations that impair enzymes involved in the breakdown of large sugar molecules called glycosaminoglycans (GAGs). This results in a toxic buildup of GAGs within cells.
SLS-005 is an intravenous (administered directly into the bloodstream) therapy that contains a sugar molecule, trehalose. This sugar molecule is able to get into the brain and activate multiple cellular processes there, including promoting proper protein folding and activating autophagy (sometimes termed the “recycling” process of the cell).
These effects are thought to reduce the accumulation of toxic molecules in the brain. Therefore, SLS-005 is currently being investigated in conditions characterized by toxic buildups in the brain, such as amyotrophic lateral sclerosis, oculopharyngeal muscular dystrophy (OPMD), and spinocerebellar ataxia type 3 (SCA3).
With the new designation, SLS-005 has been named an orphan drug in both the U.S. and Europe for Sanfilippo syndrome, SCA3, and OPMD. It has also been given fast track designation by the U.S. Food and Drug Administration for the treatment of OPMD.
Seelos is currently planning a Phase 2b/3 clinical trial to evaluate SLS-005 in people with Sanfilippo syndrome types A and B, which is being done in collaboration with the nonprofit Team Sanfilippo Foundation.
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