Tiziana Life Sciences Submits FDA Orphan Drug Designation Request for Intranasal Foralumab in Multiple System Atrophy – Tiziana Life Sciences
Tiziana Life Sciences, Ltd. announced that it has submitted a request to the FDA for Orphan Drug Designation for intranasal foralumab for the treatment of Multiple System Atrophy (MSA).
MSA is a rare, rapidly progressive neurodegenerative disorder that affects autonomic functions (such as blood pressure and bladder control) and motor control, leading to severe disability and shortened life expectancy. There are currently no FDA-approved disease-modifying therapies for MSA. The disease has a mean incidence in the U.S. of approximately 0.6 per 100,000 person-years, rising to about 3 per 100,000 in those aged 50 and older, with prevalence estimates of roughly 1.9–4.9 per 100,000 worldwide. Median survival is typically 6–9 years. Neuroinflammation and microglial activation are increasingly recognized as key contributors to MSA pathogenesis.
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. The FDA’s Office of Orphan Products Development (OOPD) aims to complete its review of an orphan drug designation (ODD) request within 90 days of receipt. If granted, the designation would provide potential benefits including seven years of market exclusivity upon approval, tax credits for qualified clinical testing, waiver of certain FDA application fees, and eligibility for protocol assistance.
Tiziana is currently evaluating intranasal foralumab in a Phase 2a open-label clinical trial (ClinicalTrials.govIdentifier :NCT06868628 ) in patients with MSA. The six-month study is designed to assess effects on microglial activation (via PET imaging), clinical outcomes, and safety. Foralumab is administered via nasal spray across eight 3-week dosing cycles. The Phase 2a trial is being conducted within the MyTrial Program at Brigham and Women’s Hospital in Boston under the leadership of Principal Investigator Vikram Khurana, MD, PhD, Tracy T. Batchelor Endowed Chair and Division Chief of Movement Disorders at Mass General Brigham.
“Multiple system atrophy remains one of the most challenging neurodegenerative diseases we face, with rapid progression and no approved therapies that alter its course. The growing recognition of neuroinflammation and microglial activation as central drivers of MSA pathology makes intranasal foralumab a particularly compelling candidate. By harnessing the immune system’s regulatory pathways in a targeted, non-systemic way, this approach has the potential to address a critical unmet need. The ongoing clinical study, which includes PET imaging of microglial activity, will provide important early insights into whether this strategy can meaningfully impact the disease process.” – Dr Khurana.
“Filing for Orphan Drug Designation for intranasal foralumab in MSA represents an important regulatory step as we advance this novel therapy for patients facing a devastating disease with no approved disease-modifying options. Foralumab administered intranasally induces regulatory T cells and modulates T-cell-driven inflammation. Its ability to reduce microglial activation offers a promising strategy to slow neuronal damage in neuroinflammatory and neurodegenerative conditions including MSA. This non-systemic approach is designed to promote immune tolerance while minimizing systemic immune suppression.” – Ivor Elrifi, Chief Executive Officer of Tiziana Life Sciences.
