Tiziana Life Sciences Files for Orphan Drug Designation with the FDA for Intranasal Foralumab for the Treatment of Multiple System Atrophy (MSA)

Orphan designation could bring tax credits and fee waivers, plus seven years of market exclusivity upon approval.

Rhea-AI Summary

Tiziana Life Sciences (Nasdaq: TLSA) has requested FDA Orphan Drug Designation for intranasal foralumab to treat multiple system atrophy (MSA). The rare, progressive disorder affects autonomic functions and motor control and has no FDA-approved disease-modifying therapies. If granted, designation could provide seven years of market exclusivity upon approval, qualified clinical-testing tax credits, certain FDA application-fee waivers and protocol assistance eligibility.

Tiziana is evaluating the candidate in a six-month Phase 2a open-label trial at Brigham and Women’s Hospital in Boston. The study assesses microglial activation—activity of immune cells in the brain—using PET imaging, alongside clinical outcomes and safety. The FDA’s Office of Orphan Products Development aims to review designation requests within 90 days of receipt.

Positive

• Minor pointOrphan Drug Designation request submitted for intranasal foralumab in multiple system atrophy.
• Minor point. Forward-looking: it has not happened yet and may not happen.Potential designation benefits include seven-year exclusivity upon approval, clinical-testing tax credits, application-fee waivers and protocol assistance eligibility.
• Minor pointSix-month Phase 2a trial underway assessing microglial activation, clinical outcomes and safety in MSA.

Negative

• Minor pointOrphan designation remains pending; potential incentives depend on the FDA granting the request.

Argus 15 min delay 1 alert

+5.69% vs previous close $1.01 last price 16.4x rel. volume Open Argus

Details

Market move: TLSA +5.69% vs previous close. Orphan drug designation filing

$0.94 – $1.04 Day Range

$128.28M Market Cap

On Sep 30, the day this news came out, the latest delayed price for TLSA is 5.69% above the previous close. The latest delayed price is $1.01. Relative volume is exceptionally heavy at 16.4x the average.

Data tracked by StockTitan Argus (15 min delayed). Upgrade to Gold for real-time data.

Key Figures

Orphan designation review period: within 90 days Potential market exclusivity: 7 years Orphan drug population threshold: fewer than 200,000 people in the United States

Orphan designation review period

within 90 days

FDA aims to complete review from receipt of the request

Potential market exclusivity

7 years

If designation is granted, upon approval

Orphan drug population threshold

fewer than 200,000 people in the United States

FDA designation eligibility definition stated in the article

Key Terms

orphan drug designation, monoclonal antibody, microglial activation, pet imaging

4 terms

orphan drug designation regulatory

"submitted a request to the U.S. Food and Drug Administration (FDA) for Orphan Drug Designation"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

monoclonal antibody medical

"a fully human, anti-CD3 monoclonal antibody"

A monoclonal antibody is a laboratory-made protein designed to recognize and attach to a specific target in the body, such as a disease-causing substance or cell. It functions like a highly precise lock-and-key tool, helping to treat or detect illnesses. For investors, companies developing monoclonal antibodies can represent promising opportunities in the healthcare sector, especially as these treatments often address unmet medical needs.

microglial activation medical

"assess effects on microglial activation (via PET imaging)"

Microglial activation is when the brain’s resident immune cells — microglia — switch from a resting, maintenance role into an alert, defensive state, similar to a neighborhood cleanup crew suddenly racing to douse a small fire. For investors, this matters because persistent or excessive activation is a hallmark of many neurological diseases, affects how drugs perform in trials, and can drive regulatory and market reactions to therapies aimed at calming or harnessing this response.

pet imaging medical

"microglial activation (via PET imaging), clinical outcomes, and safety"

PET imaging is a noninvasive medical scan that works like a molecular camera, using tiny radioactive tracers to reveal biological activity inside the body—for example metabolism, blood flow, or the presence of specific proteins. It matters to investors because PET results guide diagnosis, show whether a drug reaches its intended target and how patients respond, and therefore affect clinical trial success, regulatory approval, reimbursement decisions and demand for scanners, tracers and related services.

AI-generated analysis. How Rhea-AI works. Not financial advice.

See more from StockTitan in Google Search and AI answers. Adds StockTitan as a preferred source · opens Google

Add on Google Not now

BOSTON, Sept. 30, 2026 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, today announced that it has submitted a request to the U.S. Food and Drug Administration (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.gov Identifier: 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.

Dr Khurana commented: “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.”

Ivor Elrifi, Chief Executive Officer of Tiziana Life Sciences, commented: “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.”

About Foralumab
Foralumab, a fully human anti-CD3 monoclonal antibody, is a biological drug candidate that has been shown to stimulate T regulatory cells when dosed intranasally. At present, 14 patients with Non-Active Secondary Progressive Multiple Sclerosis (na-SPMS) have been dosed in an open-label intermediate sized Expanded Access (EA) Program (NCT06802328) with either an improvement or stability of disease seen within 6 months in all patients. In addition, intranasal foralumab is currently being studied in a Phase 2a, randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial in patients with non-active secondary progressive multiple sclerosis (NCT06292923), as well as additional Phase 2 trials in AD and MSA.

Intranasal foralumab is the only fully human anti-CD3 monoclonal antibody (mAb) currently in clinical development. Immunomodulation by intranasal foralumab represents a novel avenue for the treatment of neuroinflammatory and neurodegenerative human diseases.^[1],[2]

About Tiziana Life Sciences

Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative intranasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb currently in clinical development, has demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.

For more information about Tiziana Life Sciences and its innovative pipeline of therapies, please visit www.tizianalifesciences.com.

Forward-Looking Statements

Statements in this press release may be “forward-looking statements” within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended, that concern matters that involve risks and uncertainties that could cause actual results to differ materially from those anticipated or projected in the forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company’s current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as ‘anticipates,’ ‘expects,’ ‘intends,’ ‘plans,’ ‘believes,’ ‘seeks,’ ‘estimates,’ and similar expressions are intended to identify forward-looking statements. These forward-looking statements reflect the current beliefs and expectations of Tiziana’s management and include statements regarding the closing of the Offering. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company’s control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Tiziana’s Annual Report on Form 20-F for the year ended December 31, 2025, and other periodic reports filed with the SEC from time to time. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

For further inquiries:

Tiziana Life Sciences Ltd
Paul Spencer, Business Development, and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com

[1] https://www.pnas.org/doi/10.1073/pnas.2220272120
[2] https://www.pnas.org/doi/10.1073/pnas.2309221120

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What FDA designation has Tiziana requested for foralumab in MSA?

Tiziana has requested Orphan Drug Designation for intranasal foralumab to treat multiple system atrophy. If granted, benefits could include seven years of market exclusivity upon approval, tax credits for qualified clinical testing, certain FDA application-fee waivers and eligibility for protocol assistance.

How is foralumab administered in Tiziana’s MSA trial?

Foralumab is administered by nasal spray across eight 3-week dosing cycles. The six-month Phase 2a open-label study is registered as NCT06868628 and is being conducted within the MyTrial Program at Brigham and Women’s Hospital under principal investigator Vikram Khurana.