FDA grants incentives for development of promising new treatment for mesothelioma
An experimental anti-cancer drug currently being tested in clinical trials sponsored by the National Cancer Institutes has been granted orphan drug status by federal regulators.
BAY 94-9343 is an antibody-drug conjugate, or ADC, a new type of therapy that consists of an antibody and cytotoxic drug. Developed by Bayer HealthCare, BAY 94-9343 targets mesothelin, an antigen on the lining of the lungs, heart, and abdomen that is present at higher levels on malignant mesothelioma cells.
Bayer reports that in preclinical trials BAY 94-9343 demonstrated potent anti-cancer activity against tumors that overexpress mesothelin. The company has been recruiting patients for a Phase I dose escalation study, which evaluates the safety and tolerability of experimental drugs, since September 2011.
Orphan drugs are treatments for rare diseases that affect fewer that 200,000 people. The Food and Drug Administration's designation offers pharmaceutical companies certain incentives, such as tax breaks, aimed at helping the drug gain regulatory approval. The designation does not shorten the duration of the regulatory review and approval process, according to the FDA.
Mesothelioma is a rare malignancy that attacks the mesothelium, the protective membrane that lines lungs, heart and abdomen. More than 3,000 cases of mesothelioma are diagnosed annually in the U.S. Most patients die within 18 months of learning they have the disease.
An extremely aggressive disease, mesothelioma has proven stubbornly resistant to surgery and other conventional treatments. Only one drug, pemetrexed (sold as Alimta), has been approved by the FDA for the treatment of mesothelioma. But new therapies are on the horizon, researchers say, including at least two other orphan drugs that are currently the subject of clinical trials.
Late last year, a drug called CBP501, a “novel synthetic peptide” developed by Canbas, a Japanese company, was granted orphan drug status in the U.S. The drug increases concentrations of the chemotherapy cisplatin in cancer cells, enhancing the drug's effectiveness. Canbas recently announced the preliminary results of a Phase II trial of CBP501, which suggests the drug slows the progression of mesothelioma by blocking cancer cells from transitioning through the cell cycle.
In June, Italian researchers presented data on 57 patients who participated in a Phase II clinical trial of another orphan drug, NGR-hTNF. The results showed that patients with recurring pleural mesothelioma treated with NGR-hTNF survived five times longer than a control group.
The drug, now being tested in a Phase III trial at 40 cancer centers in Europe and the United States, is a vascular targeting