LPL deficiency treatment poised to be first gene therapy in Europe

For the first time, the European Medicines Agency (EMA) has recommended gene therapy for approval in Europe.

Alipogene tiparvovec, which treats lipoprotein lipase (LPL) deficiency, has twice been given a negative opinion by the Committee for Medicinal Products for Human Use (CHMP) and the Committee for Advanced Therapies (CAT). Now, CHMP has followed CAT's advice to recommend the therapy for approval but in a much more specific group of patients than previously applied for. As a result, alipogene tiparvovec will only be available to patients with severe or multiple pancreatitis attacks.

The decision provides the first treatment option for patients with the ultra-rare disorder who currently have to manage their condition by reducing their dietary fat to less than 20% of their caloric intake. This diet is notoriously difficult to adhere to and patients remain at risk for life-threatening acute pancreatitis, as well as increased morbidity and mortality.

Alipogene tiparvovec is an adeno-associated viral vector that contains a naturally occurring gain-of-function variant of LPL. It is administered by intramuscular injection to improve lipoprotein metabolism.

The EMA's recommendation is based on data from 27 patients treated in the Netherlands and Canada. The initial trial included 22 patients. Researchers found that while the treatment reduced triglyceride levels in the first few weeks following treatment, these changes were only transient. However, the rate of pancreatitis attacks significantly decreased from an average of 0.33 to 0.06 episodes per patient per year, when they were followed up for 1-3.5 years.

This was supplemented by a study in five other patients that specifically looked at the effect of the therapy on chylomicrons.

"The overall result is a much reduced postprandial level of newly formed, large/buoyant chylomicrons, which are thought to be the most pathogenic and causal in eliciting acute (recurrent) pancreatitis in LPL [deficiency]," said study authors André Carpentier (Centre Hospitalier Universitaire de Sherbrooke, Québec, Canada) and colleagues.

Given the rarity of the condition, patients from the trials will be monitored for 15 years. Amsterdam Medical Therapeutics (the Netherlands), which developed the therapy, will also be required to submit further detail and maintain a registry of all patients.

Tomas Salmonson, acting chair of the CHMP stated: "We have worked out a way to ensure robust and close follow-up of the quality, safety and efficacy of Glybera [alipogene tiparvovec] while giving patients who have to live with this rare disease access to a medical treatment."

The recommendation will now be sent to the European Commission for adoption of marketing authorization.

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