The Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom granted early access designation to nintedanib for the treatment of pleural mesothelioma, a major step forward for patients diagnosed with the asbestos-related cancer.
Nintedanib, marketed under the brand names Ofev and Vargatef, is a small-molecule enzyme inhibitor. In clinical trials, the drug has shown the ability to prevent mesothelioma cell growth and slow tumor spread (metastasis).
“This is potentially quite significant,” Dr. Dean Fennell, chair of thoracic medical oncology at the University of Leicester and University Hospitals of Leicester NHS Trust, told Asbestos.com. “Certainly, this drug is promising. Fantastic news.”
The U.S. Food and Drug Administration (FDA) in December 2016 granted nintedanib an orphan drug designation for mesothelioma, encouraging its use before officially approving it.
MHRA and the FDA are moving the drug another step closer to changing the long-accepted but relatively ineffective standard-of-care treatment for unresectable pleural mesothelioma.
“This is an exciting prospect of what’s to come,” Fennell said. “The drug has shown real effectiveness.”
Doctors in the U.K. and U.S. diagnose an estimated 2,500 to 3,000 combined cases of mesothelioma each year.
U.K. doctors have used nintedanib in combination with chemotherapy to treat certain lung cancers, but this recent designation as a “Promising Innovative Medicine” (PIM) expands its use significantly.
The FDA previously approved nintedanib for pulmonary fibrosis, but until a year ago it was only available to mesothelioma patients through clinical trials. The drug has worked well in relieving breathing problems by softening the lungs.
The latest U.K. designation stems from the phase II portion of an ongoing LUME-Meso clinical trial conducted at 134 sites worldwide. It is measuring the effectiveness of nintedanib in combination with chemotherapy against chemotherapy alone.
Early results of the trial include a progression-free survival of 9.4 months with the combination treatment, compared to just 4.7 months for chemotherapy alone. The overall survival benefit was 18.3 months against 14.2 months.
The clinical trial — which is still accepting new patients — has moved into phase III with an expected completion of Oct. 30, 2019. Only patients with the epithelioid subtype of mesothelioma are eligible.
There are five trial sites within the U.K. and 10 in the U.S., including:
The Barbara Ann Karmanos Cancer Institute at Wayne State University in Detroit, in conjunction with the National Cancer Institute, is conducting a nintedanib clinical trial involving recurrent pleural mesothelioma, but without chemotherapy.
Researchers at Institut Gustave Roussy in France are conducting a trial for mesothelioma involving a combination of nintedanib and pembrolizumab (Keytruda).
Oncologists involved in earlier trials with nintedanib and mesothelioma were equally impressed with the drug.
“There is a lot of excitement about this drug right now,” Dr. Rama Balaraman at Florida Cancer Affiliates in Ocala, told Asbestos.com. “It is potentially a new way of approaching this disease.”