Research and clinical trials
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The U.S. Food and Drug Administration granted orphan drug designation to ONCOFID-P for the treatment of malignant mesothelioma, a step toward another potential treatment option for patients.
ONCOFID-P is an innovative conjugated drug already in advanced clinical development for the treatment of invasive bladder cancer. The designation of mesothelioma is based on preclinical data showing high antitumor activity in in vitro and mouse models.
Fidia Farmaceutici, a multinational research company headquartered in Italy, has announced the latest US Food and Drug Administration classification, which is designed to encourage the development of new drugs, particularly for rare diseases, and help facilitate the approval process.
“I am very excited about where we are, and where this is heading,” University of Padua Professor Antonio Rosato, who led the drug’s development, told Asbestos.com’s Mesothelioma Center. “This represents an important step forward for treatment.”
Get rid of toxic side effects
The drug combines paclitaxel, a type of chemotherapy also known as Taxol, with hyaluronic acid, a naturally occurring substance produced by bacteria often used for its cushioning effect with various joint disorders.
With paclitaxel, hyaluronic acid provides binding to specific cancer cells expressing CD44, a molecule involved in proliferation and migration.
This association promotes an increase in the intracellular concentration of paclitaxel – making it more effective – and limits systemic dissemination and any resulting toxicity. ONCOFID-P, along with intrapleural delivery, should have very limited side effects.
“It’s a new concept,” Rosato said. “And what we saw was very encouraging.”
According to preclinical data, ONCOFID-P was significantly more effective in reducing mesothelioma mass than paclitaxel when used alone.
Phase 1 clinical trials coming soon
Rosato, who led the preclinical studies, serves as director of the Immunology and Molecular Oncology Diagnostic Unit and deputy scientific director at Istituto Oncologico Veneto IRCCS.
Rosato said that a phase I clinical trial for patients involving ONCOFID-P to treat pleural mesothelioma should be announced soon. The ongoing clinical trial involving invasive bladder cancer has already confirmed safety and efficacy issues.
“I don’t want patients to develop any major delusions before being tested with mesothelioma,” Rosato said. “Let’s see what the test shows. I’m a fan.
The urgent need for mesothelioma treatment
The Food and Drug Administration (FDA) usually grants orphan drug designation to products when the manufacturer requests them, provided they meet certain criteria. Grants the manufacturer exclusive and tax credits for an extended period.
The designation of orphan drugs does not guarantee future FDA approval. This does not mean that the drug is safe or effective.
Paclitaxel is approved by the U.S. Food and Drug Administration for use with lung, breast, pancreatic, and ovarian cancer. It has been used experimentally with prostate, esophageal, and bladder cancers. Some patients with peritoneal mesothelioma have found benefits from paclitaxel treatments after surgery.
Treatment of pleural mesothelioma is urgently needed. The US Food and Drug Administration (FDA) in 2020 approved the combination of Opdivo and Yervoy immunotherapy was the first new drug combination in 16 years for this cancer without definitive treatment.
“The FDA designation represents an opportunity for clinicians and patients who, to date, have not benefited from any innovative treatment, which we are proud of,” said Carlo Pizzocaro, President and CEO of Fidia Farmaceutici, in a company statement. “This shows that scientific research must not stop, and that every step forward will add hope to patients, especially those who are victims of such a serious rare disease.”
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