FDA has come up with several incentives for companies that create treatments for rare diseases such as mesothelioma. These incentives, which involve expedited movement through the FDA drug approval process or market exclusivity, are offered mostly because the audience for these therapies is so small that the drugs would probably not otherwise turn a profit.
Last week, Targovax ASA – the company developing a promising virus-based cancer drug called ONCOS-102 – announced the FDA had given the drug Fast-Track status to treat pleural mesothelioma on positive results from recent clinical studies.
Fact-Track Status Comes 2 Months After Encouraging Status Update
The welcome news of ONCOS-102 receiving Fast-Track status to treat mesothelioma comes 60 days after Targovax released its 18-month status update on cancer patients treated with virus-based therapy and chemotherapy. Patients who got the first-line protocol had a median overall survival of 18 months, compared to 14 months for patients who only got chemotherapy.
Few drugs have shown much promise to treat mesothelioma, so FDA’s move is seen as a positive sign that they hope this therapy can help patients. The Fast-Track designation is viewed as an acknowledgment and endorsement of the promise of a drug.
It is only given to drugs whose pre-clinical and clinical efficacy are viewed as possibly addressing unmet medical needs for severe medical conditions. When a drug gets Fast-Track status, it makes the regulatory review process go faster and makes it likely that the agency will review it for less time.
ONCOS-102 – Once Designated an Orphan Drug
ONCOS-102 has been viewed by medical professionals and mesothelioma patients as a drug of hope for some time. It had received Orphan Drug Designation from FDA and the EMA in Europe. Orphan Drug Designation offers a pharmaceutical company market exclusivity for even years in the US and ten in the European Union.
Targovax VP of Regulatory Affairs Ingunn Munch Lindvig, Ph.D., noted last week that obtaining Fast-Track designation is vital for the ONCOS-102 program. Fast-Track status offers the strong possibility that the drug could be a primary treatment option for solid cancer tumors with a high degree of unmet medical need.
Targovax also stated that fast-tracked products such as ONCOS-102 are more likely to get FDA priority review for a Biologics License Application and could be allowed to provide parts of the application in advance to lessen review time. An approved BLA enables Targovax to start marketing and shipping the drug.
The drug is made from adenoviruses and was made to make tumors visible to the human immune system. It is now in Phase 2 trials to complement the first-line standard of care for pleural mesothelioma patients.
How Does ONCOS-102 Work?
Researchers engineered the drug to reproduce inside cancer cells only, leaving healthy tissue and cells unharmed. They put into the virus genome a special gene coding for GM-CSF that cancer cells make when they replicate the virus. GM-CSF is a protein that sends a stimulus to the immune system.
ONCOS-102 particles enter the cancer cells and kill them. As the cancer cells die, they release cancer cell antigens that can induce an immune response. They also release new ONCOS-102 particles. This, in combination with the production of co-stimulatory GM-CSF, is a danger signal that our immune system understands and causes the death of different cancer cells.
ONCOS-102 Performed Well In Clinical Trials
A Phase 1 clinical trial called NCT01598129 reviewed the safety and efficacy of ONCOS-102 in 12 patients who were in the advanced stage of several cancers. The clinical trial results were released in the Journal for Immunotherapy of Cancer, which showed that treatment with ONCOS-102 combined with Cytoxan was safe and well-tested at the appropriate dose.
The treatment also induced an immune response at the injection site and elsewhere in the body. Most importantly, the therapy showed anti-tumor immunity with signs of a clinical benefit.
- ONCOS-102 Clinical Trial. (2021). Accessed at https://clinicaltrials.gov/ct2/show/NCT01598129
- ONCOS-102 Receives Fast-Track Status. (February 2021). Accessed at https://immuno-oncologynews.com/oncos-102/
- Targovax Receives Fast-Track Status Designation for ONCOS-102. (February 2021). Accessed at https://www.biospace.com/article/releases/targovax-receives-fast-track-designation-for-oncos-102/