The FDA has granted an orphan drug designation to the anticancer drug paclitaxel-hyaluronic acid for the treatment of malignant mesothelioma.
The FDA has granted an orphan drug designation to the anticancer drug paclitaxel-hyaluronic acid (Oncofid-P) for the treatment of malignant mesothelioma, according to a press release by Fida Farmaceutici.
Being a conjugate of paclitaxel and hyaluronic acid allows loco-regional administration of the agent. The hyaluronic acid allows for specific binding to CD44 expressing cells, increasing intracellular concentration of paclitaxel. Through this binding on the mucosal surface, the agent is concentrated in the tumor tissue, reducing the possibility of systemic diffusion. This method also allows for good tolerability.
"Patients are diagnosed in advanced-stage and experience high mortality rate after the first year. Patients have an essentially uniform prognosis across countries, and they did not benefit from significant therapeutic improvements in the last thirty years,” said Antonio Rosato, PhD professor at the University of Padua and director of the Immunology and Molecular Oncology Diagnostics Unit and Deputy Scientific Director at the Istituto Oncologico Veneto IRCCS, in a press release. "Oncofid-P represents a very important step forward for the treatment of this serious disease.”
The orphan drug designation is based on preclinical data that demonstrated the agent’s high antitumor activity. Preclinical studies have occurred in both vito and in murine models of human mesothelioma lines.
In experimental data looking at pleural mesothelioma, CD44 expressing cells are up to 70 times more susceptible to Oncofid-P compared to paclitaxel alone. When compared to paclitaxel alone, Oncofid-P was also found to be more effective in tumor mass reduction and survival. The agent was also found to be associated with low systemic exposure, meaning that antitumor activity is high, and the chance of adverse events is limited.
"The FDA designation represents an opportunity for physicians and patients who, until now, did not benefit from any innovative therapy, and makes us proud," said Carlo Pizzocaro, president and chief executive officer of Fidia Farmaceutici, in a press release. "This result demonstrates that the scientific research must not stop and that every step forward will add hope for patients, especially those who are victims of such serious rare diseases."
Human trials of Oncofid-P are currently ongoing in non-muscle invasive bladder cancer. Results on 100 patients from a phase 1/2 dose European escalation and expansion trial in this population confirmed the efficacy and tolerability of the agent. A phase 3 trial is expected to begin in Europe and the United States.