UniQure‘s Glybera has become the first gene therapy to be recommended for approval in the EU.
The European Medicines Agency‘s Committee for Medicinal Products for Human Use (CHMP) backed the therapy, which is intended to treat lipoprotein lipase (LPL) deficiency, an ultra-rare inherited disorder which leads to pancreatitis attacks.
Due to a defective gene, patients with the disorder can not produce enough LPL, an enzyme responsible for breaking down fats.
So far, management of patients with the disorder consists of a strict reduction of dietary fat to less than 20% of the daily caloric intake.
The CHMP noted that this reduction is extremely difficult to comply with and, as a result, many patients suffer life-threatening pancreatitis attacks.
Glybera uses an adeno-associated virus vector to deliver working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells.
The recommendation only covers “exceptional circumstances”, and Dutch company UniQure will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera, which the agency will review.
Amsterdam Molecular Therapeutics developed Glybera but was forced to sell up to UniQure in February 2012 after repeatedly submitting the therapy for approval with out success.
UniQure said Glybera is the first gene therapy in the western world (Ad-p53 for head and neck cancer was approved in China in 2004) to reach an “important regulatory approval milestone, culminating 40 years of research”.
Acting chair of the CHMP Dr Tomas Salmonson said, “The evaluation of this application has been a very complex process, but the use of Glybera in a more restricted indication than initially applied for, which targets the patient population with greatest need for treatment, and additional analyses by the Committee for Advanced Therapies (CAT) have added to the robustness of the data provided and allowed the CHMP to conclude that the benefits of Glybera are greater than its known risks.”
“Our established ways of assessing the benefits and risks of Glybera were challenged by the extreme rarity of the condition and also by uncertainties associated with data provided,” added Salmonson.