HQ Team
December 23, 2024: Pfizer Inc.’s combination drug to treat a type of metastatic colorectal cancer has been approved by the US drug regulator, according to a company statement.
Braftovi or encorafenib, in combination with cetuximab, fluorouracil, leucovorin, and oxaliplatin, received the green signal from the Food and Drug Administration for treating the disease in patients with a mutation in the BRAF gene that causes tumours to develop.
The mutation is detected by a test approved by the Food and Drug Administration.
The FDA’s “accelerated approval” was based on a “statistically significant and clinically meaningful improvement in response rate and durability of response in treatment-naïve patients,” according to the statement.
The accelerated approval was based on a 61% overall response rate compared to 40% in the control arm with or without chemotherapy or Pfizer’s drug during end-stage trials.
Use in first-line treatment
“Historically, treatment options have been limited and outcomes poor for patients diagnosed with metastatic colorectal cancer with BRAF mutations,” said Scott Kopetz, MD, PhD, FACP, Professor and Deputy Chair of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center and co-principal investigator of the trial.
The Braftovi drug is the first and only combination regimen featuring a BRAF-targeted therapy for the patient population, usable even in first-line treatment, he said.
“The encorafenib regimen has demonstrated high response rates that are rapid and durable. This represents an encouraging sign of continued disease control and a source of renewed hope for patients.”
The BRAF gene encodes a protein called B-Raf, which sends signals inside cells that direct cell growth. More than two decades ago, it was shown to be mutated in some human cancers and birth defects.
Next generation treatments
“Patients with metastatic colorectal cancer with a BRAF V600E mutation now have a first-line treatment option, which contains a targeted therapy specifically for a mutation that is driving their cancer,” said Chris Boshoff, MD, PhD, Chief Oncology Officer, Executive Vice President of Pfizer.
“This milestone adds to our legacy of developing innovative medicines in BRAF tumours, some of the hardest-to-treat cancers. We look forward to continuing to expand our portfolio, including the exploration of a next-generation brain-penetrant BRAF inhibitor.”
The most common adverse reactions, greater or equal to 25%, were peripheral neuropathy, nausea, fatigue, rash, diarrhoea, decreased appetite, vomiting, haemorrhage, abdominal pain, and pyrexia.
Pfizer’s application was granted priority review, used the Real-Time Oncology Review (RTOR) pilot program, and was conducted under Project Orbis, with application reviews ongoing for Project Orbis partners, including Canada and Brazil.
Third most common form of cancer
Colorectal cancer is the third most common type of cancer in the world, with approximately 1.8 million new diagnoses in 2022.
Overall, the lifetime risk of developing the disease is about one in 23 for men and one in 25 for women. In the US alone, an estimated 152,810 people will be diagnosed with cancer of the colon or rectum in 2024, and approximately 53,000 are estimated to die from the disease each year.
For 20% of those diagnosed, the disease has metastasized, or spread, making it harder to treat.
Pfizer has exclusive rights to Braftovi in the US, Canada, Latin America, the Middle East, and Africa.
Ono Pharmaceutical Co., Ltd. has exclusive rights to commercialise the product in Japan and South Korea.
Medison has exclusive rights to commercialise the product in Israel and Pierre Fabre has exclusive rights to commercialise the product in all other countries, including Europe and Asia (excluding Japan and South Korea).
