Tiziana Life Sciences seeks FDA orphan drug status for foralumab in MSA
The company filed an Orphan Drug Designation request on September 30, 2026 for intranasal foralumab in multiple system atrophy, a rare disease with no approved disease-modifying treatments.
Tiziana Life Sciences Ltd announced on September 30, 2026 that it has submitted a request to the U.S. Food and Drug Administration for Orphan Drug Designation for intranasal foralumab for the treatment of Multiple System Atrophy (MSA)1. Foralumab is described by the company as a fully human, anti-CD3 monoclonal antibody1.
MSA is characterized in the release as 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 expectancy1, and there are currently no FDA-approved disease-modifying therapies for MSA1. The company cited incidence estimates 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 to 4.9 per 100,000 worldwide1, and noted median survival is typically 6 to 9 years1.
If granted, orphan designation would provide seven years of market exclusivity upon approval, tax credits for qualified clinical testing, waiver of certain FDA application fees, and eligibility for protocol assistance1. The FDA's Office of Orphan Products Development aims to complete its review of an orphan drug designation request within 90 days of receipt1.
Foralumab is currently being evaluated in a Phase 2a open-label clinical trial (NCT06868628) in MSA patients, a six-month study designed to assess effects on microglial activation via PET imaging, clinical outcomes, and safety, with dosing delivered via nasal spray across eight 3-week cycles1. The company's overview of its pipeline states that intranasal foralumab is also being studied in a Phase 2a randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial in non-active secondary progressive multiple sclerosis (NCT06292923), as well as additional Phase 2 trials in AD and MSA1, though the release does not specify whether the MSA trial referenced here is separate from NCT06868628.
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