The Food and Drug Administration gave the go-ahead last week to accept Seelos Therapeutics’ application for a new drug to combat Sanfilippo syndrome, a rare genetic defect.
"Sanfilippo syndrome is a lysosomal storage disorder," Kathleen Buckley, a spokeswoman for the Team Sanfilippo Foundation, told the FDA Reporter. "Children are born with a genetic defect that prevents them from breaking down heparin sulfate. The storage does progressive damage to the brain and body causing the child to lose the ability to talk, walk and swallow. Life expectancy is teenage years."
According to a press release, the FDA has advised Seelos that it can go ahead with a proposed clinical trial SLS-005-201 for Mucopolysaccharidosis type III (Sanfilippo syndrome).
"Trehalose activates autophagy and we are hopeful that it will slow down or stop the progression of the disease," Buckley said. "We are hopeful that the FDA approves Trehalose if the trial is successful. Trehalose has a long safety history making it a very attractive treatment strategy if it makes an impact on the disease."
Doctors were happy to hear of the news about the clinical trial for the rare disease.
"We are very pleased to have received the acceptance letter and continue to work closely with the FDA to finalize the details of the analysis methodology for primary and secondary endpoints to demonstrate efficacy of trehalose in Sanfilippo syndrome," said Warren W. Wasiewski, M.D., F.A.A.P., Chief Medical Officer of Seelos. "Due to support and interest from families of patients with Sanfilippo, we continue to engage with additional clinical trial sites to accommodate enrollment for this trial."
"This is a great milestone for Seelos and outlines the dedication and hard work of our clinical team under the leadership of Dr. Warren Wasiewski, our CMO," said Raj Mehra, Ph.D., Chairman and CEO of Seelos Therapeutics. "We are grateful for the detailed guidance from the regulatory agency (FDA) and thank our financial supporter and collaborator, TSF, that has brought a singular focus to helping the Sanfilippo community."
The press release described Trehalose as a "low molecular weight disaccharide (.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material."
Team Sanfilippo Foundation was founded in 2008 by parents whose children were afflicted with the disease.
According to the foundation website, there are four types of Sanfillippo syndrome. And each type is named according to the enzyme that is missing.
As the disease progresses, the children lose the ability to speak, walk, eat and eventually die.
