EMA recommends first gene therapy drug for approval

Glybera offers treatment for severe or multiple pancreatitis attacks due to LPL deficiency

The European Medicines Agency (EMA) has recommended approval of Glybera (alipogene tiparvovec), the world’s first gene therapy drug, after rejecting it on three previous occasions.

The drug, manufactured by Dutch biotechnology company uniQure, is a treatment for lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks.

LPL deficiency is an extremely rare genetic disorder estimated to affect only one or two people in a million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats.

EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the marketing authorisation under ‘exceptional circumstances’. 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 as they become available.

Dr Tomas Salmonson, acting chairman of the CHMP, 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.’

He said the established ways of assessing the benefits and risks of Glybera were challenged by the rarity of the condition and also by uncertainties associated with data provided.

‘In close cooperation with the CAT we have worked out a way to ensure robust and close follow-up of the quality, safety and efficacy of Glybera while giving patients who have to live with this rare disease access to a medical treatment,’ he added.

Jörn Aldag, ceo of uniQure, said: ‘Patients with LPL 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, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPL patients and for medicine as a whole.

‘We expect final approval from the EC within 3 months after the CHMP decision.’

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