According to a September 2022 article from OncLive, the European Medicines Agency (EMA) granted Orphan Drug designation to CAN-2409. This therapy is being developed as a potential therapeutic option for those with glioma, a rare brain tumor that originates from glial cells. The Orphan designation from the EMA is not the therapy’s first designation. In fact, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to CAN-2409, in conjunction with valacyclovir, for those with glioma. 

What is CAN-2409?

Candel Therapeutics describes CAN-2409 as:

an adenovirus-based replication deficient engineered gene construct encoding the thymidine kinase gene derived from the herpes simplex virus [that] is injected directly into the tumor or target tissue. The prodrug-derived cytotoxic nucleotide analogs are designed to inhibit DNA replication and repair, leading to the death of multiplying tumor cells, and in particular of cells undergoing repair from radiation and chemotherapy damage.

Outside of glioma, Candel Therapeutics is also exploring CAN-2409 as a potential treatment option for prostate, pancreatic, lung, and brain cancers. These are being explored in various clinical trials, including a Phase 1 clinical trial evaluating CAN-2409 alongside Temodar and Opdivo. 

CAN-2409 received Orphan Drug designation from the EMA. This designation is given to therapies intended to treat, prevent, or diagnose a rare, life-threatening, and/or chronically debilitating condition. “Rare,” for the EMA, means any condition affecting no more than 5 in every 10,000 people. Incentives include marketing authorization applications, pre-authorization inspections, and reduced protocol assistance fees, among others. Learn more about this designation.

About Glioma

As described above, a glioma is a rare tumor that forms on the brain and spinal cord. Although it is considered to be rare, gliomas are also one of the most common forms of primary brain tumors. There are numerous forms and subtypes of glioma: ependymomas, glioblastomas, oligodendrogliomas, and astrocytomas (among others). Risk factors include radiation exposure and a family history of glioma. These tumors are also slightly more likely to affect males than females.

Symptoms may vary depending on tumor size, location, and rate of growth. These can (but do not always) include:

• Nausea and vomiting
• Headaches
• Problems with balance and coordination
• Memory loss and confusion
• Vision loss
• Arm, leg, and facial weakness
• Urinary incontinence
• Irritability
• Changes in mood, behavior, and personality
• Dizziness
• Seizures
• Brain function decline