Seelos Therapeutics Inc (NASDAQ:SEEL) shares raced higher on Thursday on the announcement that the biopharmaceutical company received its Orphan Drug Designation for its drug SLS-005, or Trehalose.
The drug is being developed as a treatment for patients with Type A and B versions of Sanfilippo syndrome, a childhood disorder that triggers fatal brain damage.
Orphan Drug Designation, or ODD, is given to drugs that treat a rare disease or condition. The designation allows companies developing the drug to access various incentives like tax credits for qualified clinical testing.
READ: Seelos to design Sanfilippo syndrome study of its drug Trehalose in Europe following regulatory guidance
SLS-005 was already granted ODD status by the US Food and Drug Administration (FDA) as well as the European Medicines Agency (EMA) for treatment of Spinocerebellar Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy.
New York-based Seelos also submitted an application to the FDA requesting Rare Pediatric Disease Designation for SLS-005 and is awaiting the regulator’s response.
The company recently announced it is moving forward with a Phase 2b/3 study of SLS-005 in patients with Sanfilippo syndrome types A and B after receiving written feedback from the EMA.
Based on the agency’s guidance, Seelos will design an open-label, non-placebo controlled study for the treatment of Sanfilippo patients in the European Union.
Shares of Seelos gained 20% in Thursday afternoon trading at $0.71 in New York.
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