FDA Grants Orphan Drug Designation to Experimental Acute Lymphoblastic Leukemia Drug

According to a press release from British biopharmaceutical manufacturer Autolus Therapeutics, the American Food and Drug Administration (FDA) has granted the Company’s experimental acute lymphoblastic leukemia (ALL) cell-based immunotherapy treatment AUTO3 orphan drug designation.

Orphan drug designation brings with it a host of potential benefits pending on the drug’s eventual approval by the American regulatory agency. Though the European Medicines Agency (EMA – the European equivalent of the medicinal role filled by the FDA) also grants orphan drug designation to prospective therapies, it has not yet granted AUTO3 the special status.

About Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (or ALL), like all leukemias, is a cancer of the blood and bone marrow.

ALL is the most common form of childhood cancer, and can progress rapidly and aggressively. When detected early, treatment can be especially effective – sometimes even resulting in complete remission. However, when ALL occurs in adults, it is often more aggressive and treatment yields fewer positive results.

Like many cancers, ALL begins when a faulty segment of DNA fails to signal a reproducing cell to stop dividing. This unchecked cell division leads to the formation of what can be life-threatening tumors.

About AUTO3

AUTO3 is a form of immunotherapy. Immunotherapy is a rapidly growing field of medical research that uses and modifies the body’s own defense systems to fight cancer.

Specifically, AUTO3 uses “enriched” T-cells collected from the target patient to combat ALL. A T-cell is a subtype of white blood cell that hunts down and destroys infected or cancerous cells in the body. On their own, T-cells might not be effective in fighting off ALL or other aggressive cancers. That’s why scientists “enrich” the harvested T-cells by genetically modifying them to express a surface-level antibody capable of targeting cancerous cells.

AUTO3’s orphan drug designation comes following encouraging results from a phase 1/2 study of pediatric ALL patients earlier this year. Additional trials will likely be required before final approval to compare the drug’s efficacy against existing forms of ALL treatment.

In the United States and Europe, orphan drug designation brings with it a host of advantages – including tax breaks, grant funding for the clinical trials, and, famously, a seven-year period of sales exclusivity that goes into effect upon final market approval.

Although AUTO3 has not yet been approved by the FDA or EMA, Autolus’ push to get AUTO3 an orphan drug designation shows the company is serious about developing the treatment for public use.


Acute lymphoblastic leukemia is the most commonly identified form of cancer in children. What do you think of AUTO3’s orphan drug designation? Does it give you hope for future FDA approval? Share your thoughts with Patient Worthy!

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