Decades of R&D into gene therapy is on the cusp of delivering a commercial product, with a European Medicines Agency (EMA) advisory panel recommending approval of uniQure's Glybera for a rare, inherited disorder.
The Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) in patients who suffer severe or multiple pancreatitis attacks, despite dietary fat restrictions.
That reverses an interim verdict by the CHMP in April, which concluded at the time that Glybera was not approvable.
Glybera is now indicated for a narrower group of patients than originally envisaged, while Netherlands-based uniQure has committed to providing data from a registry set up to monitor outcomes in patients treated with Glybera.
The company notes that LPLD is very rare - affecting just one or two people per million - and means that patients are unable to break down fat particles called chylomicrons in their blood plasma.
High levels of circulating chylomicrons can cause a range of complications including pancreatitis and severe abdominal pain, so LPLD patents try to adhere to an ultra-low-fat diet to try to manage symptoms.
"Patients with LPLD 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," said Jörn Aldag, uniQure's chief executive.
"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.
Glybera is administered via a one-time series of small intramuscular injections into the legs and delivers a functional LPL gene carried by an adeno-associated virus (AAV) vector, restoring the activity of the LPL enzyme in muscle cells.
If approved by the EC, as seems likely, the therapy will be administered through a series of specialist clinics in the EU. Aldag told Bloomberg in an interview that annual sales are predicted to be in the region of €50m to €300m.
It is not the first gene therapy to reach the market. That accolade belongs to Shenzhen SiBiono Gene Technologies' Gendicine for head and neck cancer, which restores p53 gene functionality and is available only in China to date.
Two other therapies have also reached the market, namely Shanghai Sunway Biotech's Oncorine (which also targets p53) which is available in China, and Epeius Biotechnologies' Rexin-G for the treatment of a broad spectrum of chemotherapy-resistant cancers, which has been launched in the Philippines.