Tiziana Requested Orphan Status for MSA Nasal Therapy

The drug candidate for multiple system atrophy aims to modulate the immune system in this neurodegenerative condition.

Updated on Sept. 30, 2026 in Alzheimer’s

Tiziana Requested Orphan Status for MSA Nasal Therapy

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Should pharmaceutical companies prioritize the development of new treatments for rare neurodegenerative diseases?

Tiziana Life Sciences has requested FDA orphan drug designation for its nasal foralumab therapy to treat multiple system atrophy. This move seeks to advance research into a condition that currently lacks any approved disease-modifying treatments.

Why it matters

Multiple system atrophy is a progressive neurodegenerative disease with a median survival of only six to nine years. Because no existing therapies can modify the disease course, new investigational pathways are critical for patients who face a limited prognosis.

A Phase 2a open-label trial at Brigham and Women's Hospital is evaluating foralumab using PET imaging to measure microglial activation alongside clinical outcomes. The study results are preliminary as the trial remains ongoing.

The players

Tiziana Life Sciences

A biotechnology company focused on developing monoclonal antibodies for autoimmune and inflammatory diseases.

Brigham and Women's Hospital

A major teaching hospital in Boston serving as a clinical research site for advanced neurodegenerative studies.

The details

Foralumab is a fully human anti-CD3 monoclonal antibody designed to promote immune tolerance through a non-systemic approach. In this trial, it is administered nasally to address the underlying neurodegenerative processes of the condition, which involves systemic inflammatory pathways.

Ongoing: Phase 2a clinical trial of foralumab.

Health Landscape

This development represents a strategic step within the framework of the FDA Orphan Drug Act to incentivize research into rare, life-limiting conditions. It addresses a significant gap in care, as there are currently no disease-modifying treatments for multiple system atrophy.

Patients and families navigating multiple system atrophy should maintain open communication with their neurologists regarding emerging trial opportunities. These developments highlight the importance of tracking research progress for conditions that remain without established clinical interventions.

The takeaway

The pursuit of orphan drug designation highlights a concerted effort to address the high mortality associated with multiple system atrophy. Patients interested in how emerging therapies might affect their care plan should consult with a specialist about current clinical trial protocols.