Two big headlines in the pharmaceutical world in the past couple of weeks; a proposal to make changes to UK legislation for patents, so that companies are not at risk from patent infringement during clinical trials for new drugs, and the formal announcement of “Glybera” as the first gene therapy to be approved in a regulated market.
Clinical trials often involve the use of a patented drug for comparison to results produced with the drug on trial. This currently produces a risk for the pharmaceutical running the trial to be sued for patent infringement by the patent holder.
The UK Intellectual Property Office has launched a consultation to set out proposals for making the UK a destination for pharmaceutical investment. Patent legislation may be adapted to shape patent infringement provisions to eliminate the risk to companies involved in UK clinical trials. The consultation will run till the 19th December this year. It will be interesting to see if the response from the companies will provide differing or similar views in how to proceed with making the UK a favoured place to research and develop new drugs.
Meanwhile Dutch company uniQure have released Glybera, a modified virus containing a functional copy of a lipid processing enzyme. Glybera has been approved by the European Medicines Agency. This viral vector can deliver the functional gene to the skeletal muscle of patients suffering from lipoprotein lipase deficiency, a disease caused by a mutation of the gene. These patients cannot absorb dietary fat from the blood and suffer from pancreatitis. This modified viral delivery method results in the virus becoming an extra chromosomal piece of DNA, rather than integrating into the human genome. Viral integration into host genomes can produce cancerous mutations. Glybera is administered by injection into the thigh muscle.
Significant investment has gone into producing Glybera, and uniQure is looking to raise $26.7 million dollars over the next few months until revenues start to come in. The company also have four other gene therapy products with approval to enter clinical trials based on similar viral delivery methods. Success for uniQure will depend on the price set for Glybera considering it should be a one off treatment. 400-500 patients are to receive Glybera, including patients in the UK.
Researchers hope that this first gene therapy approval in Europe will be the first of many, and make it easier to get the next gene therapy approval. With previous high profile failures, the success of these new clinical trials will be critical in gaining the public’s trust. But investment in gene therapy has been growing, and successful clinical trials in the US for a variety of genetic diseases would indicate that regulatory approval in the US will follow. Gene therapy is not quite so science fiction now genetically coded medicines have shown their efficacy.