Tiziana Requested FDA Orphan Status for Foralumab

The company seeks the designation to treat multiple system atrophy, a rare neurodegenerative condition.

Updated on Sept. 30, 2026 in Alzheimer’s

Tiziana Requested FDA Orphan Status for Foralumab

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Tiziana Life Sciences has filed a request with the FDA for orphan drug designation for its nasal foralumab therapy. The treatment is currently being tested as a potential option for patients with multiple system atrophy, a disease that lacks approved disease-modifying therapies.

Why it matters

Multiple system atrophy is a severe neurodegenerative disorder with limited treatment options. Orphan drug designation could accelerate development by offering tax credits and seven years of market exclusivity if the drug is approved.

Multiple system atrophy has a median survival of six to nine years. The condition affects approximately 0.6 per 100,000 person-years in the United States, with rates rising to 3 per 100,000 for individuals aged 50 and older.

The players

Tiziana Life Sciences

This biotechnology company focuses on the development of novel therapies for neurodegenerative and inflammatory diseases.

Brigham and Women's Hospital

This teaching hospital in Boston serves as the clinical research site for the Phase 2a trial of foralumab.

The details

Foralumab is a fully human anti-CD3 monoclonal antibody designed to promote immune tolerance through a non-systemic approach. Tiziana Life Sciences is currently evaluating the therapy in a Phase 2a open-label trial, which utilizes PET imaging to monitor microglial activation, patient safety, and clinical outcomes.

The Phase 2a clinical trial of foralumab is ongoing.

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The Big Picture

This request follows the framework established by the Orphan Drug Act of 1983, which provides financial and regulatory incentives to encourage the development of therapies for rare diseases. These provisions bridge the gap for conditions that often lack commercial appeal for drug developers.

Patients currently living with multiple system atrophy have no existing disease-modifying therapies available to them. Should the therapy prove successful in clinical trials and gain regulatory approval, it could offer a new, non-systemic treatment option for the condition.

The takeaway

Orphan drug status is a key regulatory milestone for companies developing treatments for rare and severe neurodegenerative disorders. The designation process remains a vital component of the clinical development pipeline for therapies lacking existing standardized treatments.