In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. Rare diseases are those affecting fewer than 200,000 Americans. Drug developers whose products receive this designation also earn a variety of benefits such as fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon drug approval. Healio recently reported that the FDA granted Orphan Drug designation to ALLO-605, an investigational CAR T-cell therapy, for the treatment of relapsed or refractory (R/R) multiple myeloma (MM).
About ALLO-605
ALLO-605 was developed by Allogene Therapeutics (“Allogene”). In the company’s own news release regarding the Orphan Drug designation, Allogene describes ALLO-605 as:
A next-generation AlloCAR T known as a TurboCar…that targets the B-cell maturation antigen (BCMA) for the treatment of patients with relapsed/refractory multiple myeloma and other BCMA-positive malignancies. ALLO-605 incorporates Allogene’s proprietary TurboCAR technology, which allows for cytokine activation signaling to be engineered selectively into CAR T cells.
Currently, researchers are evaluating ALLO-605 in a Phase 1 clinical trial. You can learn about this trial here.
What is Multiple Myeloma (MM)?
While doctors are not sure exactly what causes multiple myeloma, a form of cancer which develops in plasma cells, many myeloma cells are missing some or all of chromosome 13. Normally, plasma cells help the body fight infections. But in multiple myeloma, cancerous cells in the bone marrow crowd out healthy blood cells, causing health issues. This cancer is more common in older individuals than younger individuals. When symptoms appear, these may include:
- Nausea
- Fatigue
- Appetite loss
- Unintended weight loss
- Excessive thirst and frequent urination
- Frequent infections
- Bone pain, especially in the spine or chest
- Constipation
- Mental fogginess/confusion
- Hypercalcemia (high levels of calcium in the blood)
- Bone fractures
- Weakness or numbness in the lower extremities
