Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose) — its investigational therapy for Sanfilippo syndrome — from the Committee for Orphan Medicinal Products (COMP), a part of the European Medicines Agency (EMA).
Following this opinion from COMP, the European Commission will make a final decision on whether to grant SLS-005 this designation.
The orphan drug designation is granted to therapies that have the potential to treat diseases that affect no more than five out of every 10,000 people in the EU. If granted, the designation would make Seelos eligible for incentives such as help designing protocols for studies, reduced regulatory fees, and market exclusivity.
SLS-005 is administered intravenously (directly into the bloodstream). It contains a sugar molecule that is able to get into the brain and activate certain processes — including promoting proper protein folding and inducing autophagy (the so-called “recycling system” of the cell) — that are expected to reduce the accumulation of toxic molecules in the brain.
As such, the medication is being investigated as a potential treatment for disorders that are characterized by the accumulation of toxic molecules in the brain. For example, Sanfilippo syndrome is characterized by the toxic accumulation of glycosaminoglycans, a type of sugar molecule. Through its various biological activities, SLS-005 is thought to reduce the accumulation of glycosaminoglycans in people with Sanfilippo syndrome, thereby alleviating symptoms.
In addition to Sanfilippo, SLS-005 is also being investigated as a treatment for amyotrophic lateral sclerosis (ALS), oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3). Results from two Phase 2 clinical trials — one conducted in people with OPMD (NCT02015481) and one done in SCA3 (NCT02147886) — found the medication to be safe and well-tolerated.
SLS-005 was recently granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for Sanfilippo syndrome. It has been given orphan designations from both the FDA and EMA for OPMD, and it has also received fast track designation from the FDA for OPMD.
Seelos is currently working on multiple clinical trials to test SLS-005 in various conditions, and is collaborating with regulatory agencies like the EMA and FDA on trial design. The company is planning a Phase 2b/3 clinical trial to test 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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