Orphan Drugs

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In the United States, the Orphan Drug Act generally defines a rare disease or condition as one affecting fewer than 200,000 people. A drug or biological product may receive orphan drug designation for the prevention, diagnosis, or treatment of a qualifying rare disease or condition. Orphan drug designation provides development incentives, but it does not mean that a product has been approved by the FDA. 

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Our initial focus: Glioblastoma

 

Glioblastoma is an aggressive cancer of the brain. Standard treatment for newly diagnosed glioblastoma generally includes surgery followed by radiation therapy and chemotherapy, although treatment depends on the individual patient and tumor. Despite available treatment, outcomes remain poor, and additional research is needed (National Cancer Institute).

 

Product candidate

 

Verax and its scientific collaborators are evaluating a specialized inhaled nitric oxide product candidate, currently referred to as GlioVite™, for potential development in glioblastoma. Our goal is to determine whether the approach has a sufficient scientific and clinical rationale to support further preclinical and clinical development.

 

GlioVite™ is investigational and has not been approved by the FDA. Its safety and effectiveness have not been established. Development is subject to scientific, clinical, regulatory, manufacturing, and financing risks.

 

Vision

 

Our long-term objective is to contribute to treatment options that may help patients live longer and maintain quality of life.  Achieving that objective will require rigorous research, appropriate regulatory review, and well-designed clinical studies.

 

Other areas under evaluation

 

Verax is also assessing potential research opportunities involving:

• Hyper-IgM syndromes

• PLCG2-associated antibody deficiency and immune dysregulation (PLAID)

• Wiskott-Aldrich syndrome (WAS)

• Hyper-IgE syndromes

 

These areas are under evaluation. Inclusion on this page does not indicate that Verax has received orphan drug designation, initiated clinical development, or demonstrated the safety or effectiveness of a product candidate for these conditions.

 

Patient notice:

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Information on this website is provided for general educational purposes and is not medical advice. Verax product candidates are not currently presented here as approved treatments. Patients should discuss diagnosis, treatment, and clinical-trial options with a qualified healthcare professional.

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