FDA Grants Orphan Drug Designation to Novel Mesothelioma Treatment

The U.S. Food and Drug Administration has granted orphan drug designation to ONCOFID-P for the treatment of malignant pleural mesothelioma, a step toward another potential therapy option for patients.

ONCOFID-P is an innovative drug conjugate already in advanced clinical development for the treatment of invasive bladder cancer. The mesothelioma designation is based on preclinical data showing high antitumor activity in laboratory and mice models.

Fidia Farmaceutici, a multinational research company based in Italy, announced the latest FDA designation, which is designed to encourage the development of novel drugs, particularly for rare diseases, and help facilitate the approval process.

“I’m quite excited about where we are at, and where this is going,” University of Padua Professor Antonio Rosato, who has led the drug’s development, told The Mesothelioma Center at Asbestos.com. “This represents an important step forward for treatment.”

Eliminating Toxic Side Effects

The drug combines paclitaxel, a type of chemotherapy also known as Taxol, with hyaluronic acid, a naturally occurring substance produced from bacteria and often used for its cushioning effect with various joint disorders.

With paclitaxel, the hyaluronic acid provides binding to specific cancer cells expressing CD44, a molecule involved in proliferation and migration.

This binding promotes increased intracellular concentration of the paclitaxel – making it more effective – and limits the systemic diffusion and any resulting toxicity. Combined with its intrapleural delivery, ONCOFID-P should have very limited side effects.

“It’s a new concept,” Rosato said. “And what we’ve seen has been very encouraging.”

According to preclinical data, ONCOFID-P was dramatically more effective in mesothelioma tumor mass reduction than paclitaxel was when used alone.

Phase I Clinical Trial Coming Soon

Rosato, who led the preclinical studies, serves as director of the Immunology and Molecular Oncology Diagnostics Unit and deputy scientific director at the Istituto Oncologico Veneto IRCCS.

Rosato said a phase I clinical trial for patients involving ONCOFID-P for the treatment of pleural mesothelioma should be announced soon. The ongoing clinical trial involving invasive bladder cancer already has confirmed the safety and efficacy issues.

“I don’t want patients to get any grand illusions before testing with mesothelioma,” Rosato said. “Let’s see what the testing shows. I’m encouraged.”

Urgent Need for Mesothelioma Treatment Advances

The FDA typically grants the orphan drug designation to products when requested by the manufacturer, provided it meets certain criteria. It gives the manufacturer tax credits and exclusivity for an extended period.

Orphan drug designation does not guarantee future FDA approval. Nor does it mean the drug is safe or effective.

Paclitaxel is approved by the FDA for use with lung, breast, pancreatic and ovarian cancers. It has been used experimentally with prostate, esophageal and bladder cancers. Some patients with peritoneal mesothelioma have found benefits from paclitaxel treatments after surgery.

There is an urgent need for treatment advances for pleural mesothelioma. The FDA’s approval in 2020 of the Opdivo and Yervoy immunotherapy combination was the first new drug combination in 16 years for this cancer with no definitive cure.

“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 CEO of Fidia Farmaceutici, in a company statement. “This 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.”

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