Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation

Seelos Therapeutics has recently received the Rare Pediatric Disease Designation for their Sanfilippo syndrome treatment, trehalose. This designation is an incentive for companies to make therapies for conditions with an unmet need, as it awards priority review vouchers. This news was not the only big announcement for Seelos; they have recently added a new member to their board of directors as well.

About Sanfilippo Syndrome

Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a disease in which the body cannot break down long chains of sugar molecules due to a lack of enzymes. These chains then accumulate, causing the symptoms of Sanfilippo syndrome. This syndrome comes in four forms: A, B, C, and D, with A being the most severe.

The onset of symptoms typically occurs about a year after birth, but they are most severe between the ages of two and six. Symptoms include:

  • Developmental issues
  • Behavioral and personality issues
  • Coarse facial features
  • Diarrhea
  • Sleep difficulties
  • Stiff joints that may not extend fully
  • Walking problems

This syndrome is inherited in an autosomal recessive pattern, meaning both parents must pass down the mutated gene in order for a child to be affected.

Doctors will diagnose Sanfilippo syndrome through a urinalysis, blood or skin sample, or genetic testing. Enzyme replacement therapy has been shown to be effective in other forms of mucopolysaccharidosis , but not this type. Treatment is symptomatic and supportive.

Seelos’ Announcements

Trehalose, also known as SLS-005, is a treatment for types A and B of Sanfilippo syndrome. This announcement comes after the FDA granted it the Orphan Drug Designation, which offers benefits like tax incentives.

The next step for Seelos and trehalose is to develop a clinical trial in Europe. The European Medicines Agency is working with Seelos in the hopes that this drug will one day be available across the world.

This announcement is paired with the addition of Judy Dunn to the board of directors at Seelos Therapeutics. She has ample experience in this field, as she has previously worked at Pfizer and Roche. She brings her knowledge of psychiatry and neurology with her, and Seelos is ecstatic to have her.

Seelos is very excited for both of these announcements. Hopefully trehalose will continue to do well in trials, allowing it to better the lives of those with Sanfilippo syndrome.

Find the source article here.


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