NeuroThera Seeks FDA Orphan Drug Status for SCI-110

NeuroThera Labs' decision to engage a government affairs firm alongside its FDA Orphan Drug Designation application for SCI-110 signals how clinical-stage CNS developers are treating regulatory strategy and federal stakeholder engagement as parallel workstreams, not sequential ones. The SciSparc subsidiary filed its designation pursuit for SCI-110, a cannabinoid-based oral candidate targeting adult Tourette Syndrome, a condition the FDA classifies as rare given its U.S. prevalence below the 200,000-patient threshold.

To support the pathway, NeuroThera has retained RK Strategies, Inc., a Washington-based government and public affairs firm founded in 2017 with a healthcare and pharmaceutical focus. The engagement is structured to build visibility among federal stakeholders and shape the policy environment around market access, a move that reflects the increasing complexity of securing orphan status for CNS indications where clinical endpoints and prevalence data require active interpretation before agency reviewers.

SCI-110 combines dronabinol with palmitoylethanolamide (PEA) in a single oral dosage form, designed to address both tic reduction and comorbid symptoms in adult TS. The candidate recently entered a Phase IIb program running concurrently at three academic sites: Yale Child Study Center, Hannover Medical School, and Tel Aviv Sourasky Medical Center. That multinational footprint carries regulatory implications, particularly around data harmonization and the applicability of non-U.S. site data to an FDA submission package.

Phase IIa results provide the clinical foundation: SCI-110 achieved an average tic reduction of 21% as measured by the Yale Global Tic Severity Scale Total Tic Score. For QA and regulatory leads tracking the program, the Phase IIb design will need to demonstrate whether that signal holds across a larger, geographically diverse population before the designation application can be reinforced with robust clinical evidence.

Orphan Drug Designation, if granted, would confer seven years of U.S. market exclusivity, eligibility for tax credits on qualified clinical trial expenses, and waiver of certain PDUFA application fees. For a clinical-stage company operating across two exchanges, those incentives carry material weight on development timelines and capital allocation decisions.

The Phase IIb readout will serve as the next measurable checkpoint for both the designation application and NeuroThera's broader regulatory trajectory in adult Tourette Syndrome.

Source: GlobeNewswire via SciSparc Ltd. press release, 2 September 2026.