European Union Approves Gene Therapy Drug

The drug Glybera is now available for commercial consumption throughout the European Union. Glybera is intended for use in lipoprotein lipase (LPL) deficiency patients, but offers promise for those suffering from other rare and aggressive illnesses. Mesothelioma attorneys hope that this new development will offer a successful treatment option for rare disease victims nationwide.

Mesothelioma is an extremely evasive disease, and much research supports that newly discovered inherited biomarkers may aid in groundbreaking gene therapy developments. In 2012, Dr. Harvey Pass of NYU Medical Center found a biomarker that is significantly elevated in mesothelioma victims. Those patients with this specific biomarker are potential candidates for gene therapy, and would greatly benefit from such treatments. Another recent discovery introduced a new stem cell drug targeting tumor suppressor genes into American and European markets for clinical trials, and is now on the FDA’S Fast Track Program.

Glybera was approved as an orphan drug by the European Commission in 2004. Orphan medicine is defined as those treatments seeking to cure or manage rare diseases, which often require grants and fast track status by government agencies. Gene therapies are developed to cure genetic diseases by replacing, mutating, or destroying defective genes. LPL deficiency does not allow the body to properly break down fats, and is caused by an inherited defective gene. Glybera delivers working copies of the defective gene into muscle cells to enable gene production.

BBC News reports that the evaluation of the Glybera application was a very complex process, involving two separate committees and one previous refusal in 2011. The two councils re-examined the drug later that year, when the Committee for Drug Agency drafted a positive opinion letter, which was ultimately approved in June 2012. Glybera was developed by Mr. Aart Brouwer of the Netherlands, and is now being sponsored by Amsterdam Molecular Therapeutics.

This is not the first gene therapy to be approved globally, however. In 2004, China became the first country to sanction a gene therapy drug. Gendicine is now the world’s first commercially produced gene therapy, after five years of clinical trials and $9.6 million in development costs. The initial results of the drug are impressive, boasting a 64% complete tumor regression and 32% partial regression rate in patients with head and neck squamous cell carcinoma.

Due to its enormous population, the availability of clinical treatment for patients with cancer and other life-threatening illnesses in China is decidedly less than in the United States. For this reason, patients tend to favor one-time therapies, such as Gendicine , instead of more conventional treatments. Despite wariness from regulatory authorities in other countries, the prediction that Western countries would soon thereafter approve gene therapies rang true. Other pharmaceutical companies, such as Introgen in France, are developing similar cancer-specific gene therapies and in the final stages of clinical trials. The councils will review Glybera patient outcomes as they become available. This decision ushered in a new era for rare disease communities, which affect more than 30 million people worldwide.

In the United States, the Orphan Drug Act designates therapies based on the seriousness of the condition it’s targeting, the existing alternative methods for diagnosis, prevention, and treatment, and the rarity of the condition. Mesothelioma drugs are often assigned as orphan medicinal products, such as Amatuximad, which targets a protein that is often over-expressed in mesothelioma patients. Asbestos exposure attorneys are hopeful that the commercial availability of gene therapies in Europe and Asia will promote growth in this field and give promise to mesothelioma and lung cancer sufferers around the world.

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