Shire readies Gaucher drug Vpriv

Shire Pharmaceuticals says it has already manufactured enough of its Vpriv to treat around 400 Gaucher disease patients and could increase that to 'several hundred' in 2010.

Meanwhile, the company expects to start production at its new large-scale facility for the product in Lexington, USA, in the middle of 2010.

Vpriv's development for Gaucher disease was accelerated by the supply problems facing rival drugmaker Genzyme, which through a large proportion of 2009 was wrestling with contamination problems at a plant used to make its own Gaucher drug Cerezyme (imiglucerase).

Faced with acute shortages, the FDA asked Shire to make Vpriv (velaglucerase alfa) available on compassionate-use grounds midway through 2009 - ahead of its approval - prompting Shire to swiftly press ahead with a full marketing application, several months ahead of schedule.

The dossier was accepted for review by the FDA towards the end of last year with a decision deadline of February 28.

In parallel, Shire accelerated the investment programme at the Lexington site on large-scale manufacturing capacity for Shire's human genetic therapies (HGT) business, including Vpriv.

The plant will initially concentrate on Vpriv production in order to meet the expected hike in demand if full marketing approval is granted by the FDA later this month, but can also be used to make other products in the Shire HGT portfolio.

On a conference call to discuss the firm's 2009 results, Shire HGT head Sylvie Gregoire noted that velaglucerase alfa is currently being made at the company's plant in Cambridge, Massachusetts.

Additional growth supported out of the new large-scale Lexington facility which will use up to four 2,000-litre bioreactors and relies extensively on single-use, disposable manufacturing components to reduce the risk of contamination.

The FDA recently completed pre-approval inspection of the VPRIV manufacturing facilities, which was "an important milestone in the review and approval process" for the drug, according to a Shire press statement.

The company also said it plans to price Vpriv at a wholesale acquisition cost of $1,350 per 400 unit vial, around a 15% discount to Cerezyme.