European regulators have slammed on the breaks and delayed an approval decision for Amsterdam Molecular Therapeutics' Glybera, a gene therapy delivered by an altered virus for patients with a rare condition that leads to recurrent pancreatitis.
This is where things get dicey. In delaying its decision, the European Commission wants more information first from the European Medicines Agency's Committee for Human Medicinal Products, which recommended against approving the drug in June, in part due to risk-related concerns. In an official statement, the Dutch company said it was "encouraged" that regulators want more data before making a decision, though regulators haven't set a final timeline for ruling on the matter.
"The ongoing debate shows that the authorities are determined to find the best process of dealing with such innovative treatments and technologies," AMT CEO Jörn Aldag said in a statement. In the meantime, the company said it is pursuing partnerships for its other treatments and slashing jobs by about half, based on the CHMP recommendation to decline Glybera's approval.
Glybera is designed to treat patients who face lipoprotein lipase deficiency, a rare inherited condition that can lead to dramatically higher levels of fat in the blood and repeated pancreatic inflammation. Those suffering from the condition have no other treatment option. Glybera is delivered through a single series of "small intramuscular injections" in the legs, according to the company's website. The drug carries a healthy LPL gene into the body in an altered adeno-associated virus that is drawn to muscle cells, the company explains. Muscle cells help build healthy LPL production.
- here's the release