European Agency Recommends Gene Therapy Approval
July 21, 2012

European Agency Recommends Gene Therapy Approval

redOrbit Staff & Wire Reports - Your Universe Online

A European Union agency for the evaluation of medicinal products is recommending the approval of a gene therapy treatment for a rare disease, which would be the first time a treatment of this kind has received the regulatory go-ahead for use in the West, according to various reports published Friday.

A statement released by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) officially recommended the authorization of Glybera (alipogene tiparvovec) for use throughout all EU member states. Glybera, which is manufactured by the Dutch firm uniQure, is used to treat lipoprotein lipase deficiency (LPLD), an extremely rare disorder that prevents people from digesting fat.

It had previously been rejected by the EMA on three occasions, but was approved at the behest of the European Commission, the Associated Press (AP) explained. Furthermore, the agency recommended approval under strict conditions, including requiring uniQure to create a registry in order to track patients using the experimental treatment, which had only been tested on 27 patients during previous clinical trials.

The recommendation requires final approval from the European Commission, but as Thomas H. Maugh II of the Los Angeles Times points out, the Commission "generally follows the recommendations of the agency, and if it does so this time, the product could be available in all 27 members of the European Union by the end of the year."

If Glybera does indeed receive approval, it will become the first gene therapy to be made available outside a clinical trial in either Europe or the United States, Guardian reporter Ian Sample said. In 2004, a gene therapy designed to treat cancer was approved in China, but the prospect of this type of treatment becoming widely available outside of the Asian market has created excitements amongst experts.

"This is a watershed moment. Gene therapy holds incredible promise for people, especially those with rare diseases," former FDA Director of Orphan Products Development Tim Coté, currently an independent consultant at the Keck Graduate Institute in California, told Sample. "This is not an isolated example, this is the beginning of something. It paves the way for the approval of other treatments of this kind."

"There are about 3,000 of these rare genetic diseases out there waiting for a cure and this is the first one to come through. If you ask me, gene therapy is the way we're going to cure those 3,000 diseases," he added.

Essentially, gene therapy replaces problematic genes with healthy copies, and while the process can be easily explained, it has been far harder to master, Sample said. Several early forms of the treatment proved ineffective as they were unable to produce enough copies of the correct cell types, and some even created the incorrect types of genes, in some cases resulting in patient death.

LPLD, which affects approximately one or two out of every million individuals, occurs when a person's body lacks an enzyme that breaks down chylomicrons, or large fat-carrying molecules that enter the circulation following a meal. The deficiency of the lipoprotein causes the chylomicrons to accumulate, leading to whitening of the blood, the blocking of smaller blood vessels, and inflammation of the pancreas.

"Glybera is an active form of lipoprotein lipase that is injected into the patient's legs in a series of shots at one sitting," Maugh explained, adding that clinical trials conducted "by Dr. Daniel Gaudet of the University of Montreal show that the injections are long-lasting and provide at least partial control of lipoprotein lipase deficiency with no apparent adverse effects. Bouts of pancreatitis are fewer and less severe following treatment."

UniQure called the ruling a "major breakthrough" for LPLD patients, and CEO Jorn Aldag told BBC News Health and Science reporter James Gallagher, "Patients with lipoprotein lipase deficiency are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care."

"Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection," he added. "Restoring the body's natural ability to break down fat particles in the blood, in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level."