European Regulators okay first gene therapy drug by Amina Shakrullah
Almost 20 years ago the first experiment to repair the faulty genes was carried out. The experiment was a breakthrough in the field of drug discovery. Since that time scientists and drug companies are working hard for the practical application of gene therapy. Now, the approval has been given by the European regulators, to the first gene therapy drug of the western world. The approval of the gene therapy drug by European Medicines Agency (EMA) is a big achievement for the drug discoverers of uniQure, a Dutch Biotech company. This gene therapy drug is a potential lifeline for the patients suffering from an ultra-rare genetic disorder lipoprotein lipase deficiency LPLD.
However, it is too late for the investors of Amsterdam Molecular Therapeutics (AMT) a previously listed firm. Since the rejection of Glybera medicine by AMT and because AMT cannot support itself financially, AMT was taken private by uniQure in April. Two largest shareholders of uniQure are Forbion Capital Partners and Gilde Healthcare. Both companies are the leading life sciences venture capital companies in the Netherlands.
Patients suffering from LPLD have lost the ability to handle fat particles in their blood plasma. These patients hesitate from taking a regular meal as it results in acute inflammation of the pancreas. The LPLD disorder may lead to acute pancreatitis and even death. It is also estimated that the disorder does not affect more than one or two persons in a million. So, it was quite challenging to win the approval for Glybera the first gene therapy drug against LPLD. Because of the rare occurrence of the disorder company got just 27 patients for clinical trials. Initially, the European agency has been hesitant to approve the drug just because of the thin number of evidences. According to a London based watchdog the drug is accepted for approval because there are sufficient numbers of evidence to give a green signal for the approval of drugs to treat the worst affected patients.
The chief executive of uniQure said in an interview, it is only difficult to get the first approval either from EMA or FDA but we have achieved this goal. This approval will help to unlock the potential of gene therapy. The idea of disease treatment by the replacement of a defective gene with a working copy of that gene got its credibility in 1990. As in 1990 the first successful clinical trial for first gene therapy was carried out against a rare disorder, Severe combined immunodeficiency SCID. The disease is also known as “bubble boy disease” and patients suffering from the disease have a defective immune system so they cannot survive with the infection and most of them die in their childhood.
In 1999 an Arizona teenager died during a gene therapy experiment and in 2002 two French boys suffering from SCID developed leukemia. These two events were the major setback in the field of gene therapy. In 2003 Shenzhen SiBiono Gene Tech a china based company got the approval for a gene therapy drug against head and neck cancer. Until now, no product has been approved in Europe or United States. Recently, some world’s leading pharmaceutical companies are also exploring gene therapy. For example, GlaxoSmithKline has signed a deal with Italian researchers in 2010 for the development of a therapy against SCID.
The acting chairman of the EMA’s committee for medicinal products for human use said, the evaluation of the first gene therapy drug Glybera was not an easy task. He said that experts have decided to recommend the drug only to the patients which have the greatest need for treatment. The uniQure company is also looking forward to get the regulatory approval for Glybera in other markets like the United States and Canada.
-Amina
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