Dr Nghiemphu on How the NCCN Inclusion of Brigatinib Expands Access for NF2-Related Schwannomatosis

“As an oral agent that is well tolerated, [brigatinib] can easily be the first medical agent that patients can try to control their tumors.”

P. Leia Nghiemphu, MD, a professor of clinical neurology, director of the Neuro-Oncology Clinical Service, and clinical director of the UCLA Neurofibromatosis Program at UCLA Health, discussed the significance of the inclusion of brigatinib (Alunbrig) in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for central nervous system cancers for the treatment of patients with NF2-related schwannomatosis.

Because NF2-related schwannomatosis is so rare, it is difficult to conduct a clinical trial that can lead to the FDA approval of a therapy for this disease, as pivotal trials usually require a randomized setting or a large patient population, Nghiemphu explained. A smaller trial, such as the phase 2 INTUITT-NF2 study (NCT04374305) can treat a small number of patents patients and therefore be completed and identify responses in a quicker time frame, but that caliber of trial cannot support the FDA approval of an agent, meaning the investigated drugs can only be used off-label for patients with NF2-related schwannomatosis, she noted.

The inclusion of brigatinib for this indication in a resource like the NCCN Guidelines demonstrates that this agent is an accepted therapy for patients with NF2-related schwannomatosis according to the NCCN, Nghiemphu said. The guidelines are assembled by experts in nervous system tumors, who have agreed there is strong evidence that the agent can help this patient population, she noted. Now that brigatinib is included in the guidelines, oncologists can approach insurance companies to request approval for patients with NF2-related schwannomatosis to receive the agent, according to Nghiemphu.

As an oral agent that is relatively well tolerated, brigatinib can easily serve as the first medical therapy patients use to try to control tumors that are growing because of NF2-related schwannomatosis, Nghiemphu said. She added that data from the study also showed a reduced tumor growth rate once patients started receiving the agent. Because NF2-related schwannomatosis is a condition in which tumors grow slowly over time without causing many symptoms until they become very large, slowing that growth could help patients avoid additional surgery for their tumors and improve their quality of life, she explained.