US regulators have issued a green light for Genzyme’s Gaucher disease capsules Cerdelga (eliglustat), offering patients the first oral first-line treatment for the rare genetic disorder.

Following a priority review, the US Food and Drug Administration gave its blessing for the drug’s use for the long-term treatment of adults with Gaucher disease type 1, which occurs in around 1 in 50,000 live births.

However, it has stipulated that the approval does not stretch to the small number of patients who metabolise Cerdelga more quickly (or at an undetermined rate), as detected by an established genetic laboratory test, because they may not achieve adequate concentrations of the drug for it to have a therapeutic effect.

Cerdelga is widely expected to become the market leader of Gaucher treatments because, as a twice-daily oral therapy, it offers a more convenient alternative to Genzyme’s current standard-of-care treatment, the enzyme replacement therapy Cerezyme (imiglucerase), which is given via lengthy and regular intravenous infusions. 

New approach

It also offers a different treatment approach to enzyme replacement therapies by slashing the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher.

The drug will also be vying for market share with Shire's Vpriv (velaglucerase alfa) and Pfizer/Protalix Biotherapeutics' Elelyso (taliglucerase alfa), though these are both injected. Actelion also has an oral offering on the market for type I Gaucher - Zavesca (miglustat) - but this is only used in people who cannot be treated with enzyme replacement therapy and poses no significant threat its potential.

Cerdelga, which also received orphan drug designation from the FDA, is expected to hit pharmacy shelves within a month, the firm said. The drug is also currently under review in Europe and elsewhere.