FDA Designation of ONCOFID®-P as Orphan Drug

Italian pharmaceutical company, Fidia Farmaceutici, announced last month that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for one of its drugs to treat malignant mesothelioma. 

The anti-cancer drug, called ONCOFID®-P, will be designated to treat advanced forms of pleural mesothelioma. The drug has already demonstrated potent efficacy and tolerability among patients with advanced non-muscle invasive bladder cancer.

Breaking Down The Orphan Drug Combination

Oncofid-P is a combination of chemotherapy drug paclitaxel and hyaluronic acid. Researchers believe the combination may significantly slow the progression of mesothelioma tumors. Data from the drug’s treatment of other forms of cancer in clinical settings show that it is ideal as a loco-regional treatment for mesothelioma.

Researchers look forward to studying the innovative combination of the anti-cancer drug and hyaluronic acid in a clinical setting to treat pleural mesothelioma because of high anti-tumor activity in preclinical testing. The hyaluronic acid helps the paclitaxel component bind to cancer cells and penetrate the disease. Components of the hyaluronic acid allow the drug to concentrate in tumor tissue while avoiding healthy cells.

Mesothelioma is a rare and aggressive cancer that usually affects the lungs but can occur, in even rarer cases, in the thoracic cavity or abdomen. The onset of the cancer is caused by exposure to asbestos. 

The FDA has only approved Oncofid-P for orphan status, meaning it still needs to be tested for safety and efficacy among mesothelioma patients in a clinical setting before becoming available for public use. 

“The FDA designation represents an opportunity for physicians and patients who, until now, did not benefit from any innovative therapy, and makes us proud,” Carlo Pizzacaro, President and CEO of Fidia Farmaceutici, stated in the press release.

Moving Through the Clinical Trial Process

Preclinical studies are still underway to complete the Pharmacol-toxicology profile that researchers aim to submit to regulatory authorities by 2022.

In previous clinical settings, Oncofid®-P has been administered intravesically (loco-regionally) to treat non-muscle invasive bladder cancer. The phase 1 study evaluated the minimum effective dose. The drug combination was also tested in a phase 2 study in low-grade papillary bladder cancer. 

Out of 100 patients in the European study, results indicated high efficacy in terms of tumor shrinkage and tolerability of the Omcofid-P among cancer patients. We can expect a phase 3 study to begin soon in the U.S and Europe for further safety testing.

The mesothelioma study of this combination is led by the University of Padua professor Antonio Rosato, who is also the Director of the Immunology and Molecular Oncology Diagnostics Unit and Deputy Scientific Director at the Istituto Oncologico Veneto IRCCS. 

Rosato, who developed the concept of the loco-regional application for Oncofid-P and conducted the preclinical trials, stated “Oncofid-P represents a very important step forward for the treatment of this serious disease. Patients are diagnosed in [advanced stages] and experience a 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.”

New Mesothelioma Treatment Options Continuously Need Exploring

Researchers say scientific research on new treatment options in hospitals across the nation for mesothelioma patients must not stop. Clinical trials are a crucial step in gaining FDA approval. Mesothelioma is often diagnosed in advanced stages when specific life-saving treatment options such as surgery are not available. 

“The FDA designation represents an opportunity for physicians and patients who, until now, did not benefit from any innovative therapy, and makes us proud. 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,” said Carlo Pizzocaro.