Orphan Drugs Gain Unprecedented Attention from Manufacturers

The Orphan Drug Act applies to pharmaceuticals developed to treat rare diseases (affecting fewer than 200,000 Americans). Recently, rare diseases such as mesothelioma are gaining significant attention from drug manufacturers, as more than a quarter of the new drugs approved by the FDA in 2012 were granted orphan drug status. Mesothelioma attorneys are hopeful that the rapidly expanding orphan drug market will lead to significant developments in mesothelioma treatment.

In late 2012, Morphotek, Inc., announced that the FDA granted orphan drug designation to its cancer drug amatuximab for treatment of malignant pleural mesothelioma. Orphan status is reserved for drugs that have the potential to safely and effectively treat, diagnose, or prevent rare diseases or disorders. Under the Orphan Drug Act, the designation allows Morphotek certain incentives, including tax credits, development product user fee waivers, and a seven-year period of exclusive marketing.

Amatuximab is an anti-body drug that targets the glycoprotein and mesothelin cell surface, which is over-expressed in a number of cancers, including mesothelioma, ovarian, and pancreatic. Researchers at both the National Cancer Institute and Johns Hopkins University have validated mesothelin as a potential treatment target.

Amatuximab is considered an immune-based therapy, and lab studies have shown that the drug helps the immune system kill cells that have high levels of mesothelin. More research is needed, however, to determine levels of safety and efficacy. The National Cancer Institute is sponsoring one clinical trial to test amatuximab, and is currently accepting participants.

Earlier in 2012, Japan’s CanBas Co. announced that the FDA granted orphan drug designation to its CBP501, a drug intended for use in conjunction with chemotherapy treatment. CBP501 was specifically designed for malignant mesothelioma patients, and acts on numerous pathways related to DNA damage and cell cycle repair. As stated, it is used in combination therapy for first-line cisplatin and pemetrexed treatment. CanBas also reported that CBP501 expressed the ability to re-sensitize tumor cells that have grown resistant to cisplatin, a common chemotherapy drug, and that Phase I studies demonstrated promise for efficacy in treating ovarian cancer as well as mesothelioma.

CBP501 allows toxic platinum, administered by the chemotherapy drugs, to build up in tumor cells through enzyme modulation. This prevents tumor cells from repairing themselves, and renders resistant cells more sensitive.

Thompson Reuters is reporting that top orphan drugs, such as CPB501 and amatuximab, are as economically viable as more commercial drugs, contrary to prior belief. With an average of 250 new rare diseases identified per year, orphan drug development is in fact in the best interest of pharmaceutical companies. The orphan drug market is currently experiencing an annual growth rate of nearly 26%, while the non-orphan drug market growth remains at just over 20%. At the close of 2011, the global orphan drug market was estimated at $50 billion.

Unfortunately, the cost of these new orphan drugs is very high, and some would argue, arbitrary. Because the population of patients using these drugs is relatively low, pharmaceutical companies believe they can needlessly hike up prices. Healthcare experts around the globe, however, are calling on these companies to base prices more on realistic future profit estimates, rather than on the financial attractiveness of exploiting patients looking for treatment.

Mesothelioma attorneys affirm that orphan drugs are an important part of the global pharmaceutical industry’s future. If you or someone you love is suffering from mesothelioma as the result of exposure to asbestos, it is important you contact a lawyer for a free legal consultation. We hope that the velocity of change in research and development of rare diseases spurs an increase in treatments available to patients.