FDA Grants Breakthrough Therapy Designation to Omburtamab for Metastatic Neuroblastoma

Editor’s note: When the original version of this story was published in June 2017, the drug was named burtomab. The name was changed to omburtamab in December 2017.

Great strides have been made in the treatment of neuroblastoma, a rare cancer of nerve tissue that usually occurs in children and typically arises in the abdomen. However, with current therapies, the aggressive form of this cancer will spread, or metastasize, to the brain. At that point, it becomes nearly impossible to control.

Now, there is hope for people facing this devastating situation. A drug created at Memorial Sloan Kettering has reached an important milestone toward becoming a treatment option for patients. The FDA has granted the drug, omburtamab, Breakthrough Therapy Designation for the treatment of neuroblastoma that has spread to the brain.

Omburtamab is a monoclonal antibody that binds to the surface of neuroblastoma cells. When it is linked to a radioisotope — a version of a chemical element that emits radiation — it becomes a drug suitable for a treatment known as radioimmunotherapy. It is injected into the spinal fluid and delivers precision liquid radiation to strike the cancer cells dead.

Omburtamab was created and tested under the name 8H9 by MSK physician-scientist Nai-Kong Cheung, who heads MSK’s Neuroblastoma Program. The drug has been licensed by MSK to Y-mAbs Therapeutics.

“Now, we have children living for years after receiving 8H9,” Dr. Cheung says. “This treatment is evidence-based and offers real hope and even cure that was previously unthinkable. I’m so glad the FDA has granted the Breakthrough Therapy Designation, since there is no approved drug to treat neuroblastoma that has spread to the brain.”

The designation was given to the drug on the basis of a clinical study of 105 children with metastatic neuroblastoma who had all been treated at MSK since 2003. Those who received it in addition to conventional treatment with chemotherapy and radiation survived significantly longer than children who never received the drug. Celebrating the ten-year cancer-free milestone is now a reality for those first recruits.

The FDA grants Breakthrough Therapy Designation when early clinical data indicate that a drug may demonstrate substantial improvement over existing therapies. The designation is intended to speed up the review process of these drugs.

In addition to demonstrating the drug’s effectiveness, the trial also showed it to be safe. The approach targets the cancer cells with precision, while sparing the surrounding brain tissues from damage. This is especially important for pediatric patients because more conventional treatments, especially external beam radiation therapy, can cause learning deficits and developmental delays in young children.

Pediatric oncologist Kim Kramer, who led the clinical study, says the results of the trial — and possible approval of omburtamab by the FDA — could change the course of the disease at this late stage. Doctors at other institutions are beginning to take notice and express interest in getting this therapy to their patients — which is now routinely given in the outpatient Pediatric Day Hospital at MSK — to maximize their quality of life.

“We have met with several colleagues in the United States and Europe to help facilitate a formal collaboration that will permit omburtamab to be administered at their centers,” Dr. Kramer says. “It’s super exciting to move this forward beyond the success that we’ve had with the drug at MSK, and to enable children throughout the world the chance to receive this lifesaving treatment.”

Nai-Kong Cheung has received commercial research grants from Y-mAbs Therapeutics and is a part owner of the company. Dr. Cheung is the inventor and owner of issued patents licensed by MSK to Y-mAbs.