Shire's single-use MA plant receives US FDA approval to make VPRIV

By Dan Stanton

- Last updated on GMT

VPRIV, approved in over 40 countries as a therapy for Gaucher's disease
VPRIV, approved in over 40 countries as a therapy for Gaucher's disease

Related tags: Food and drug administration, Shire

The US Food and Drug Administration (FDA) has approved a second facility for Shire to manufacture its Gaucher disease drug VPRIV.

VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease, was approved by the FDA in Febrauary 2010 and is produced by Shire from its Alewife, Massachusetts facility.

Though the firm has pumped $200m (€146m) into manufacturing infrastructure and technology for the drug, a second Massachusetts site in Lexington has only just been granted approval to make it after addressing a Complete Response Letter (CRL) issued by the FDA in 2012.

“The FDA’s CRL addressed analytical and manufacturing procedures related to the operation of the 400 Shire Way [Lexington] facility for VPRIV,”​ Shire spokesperson Jessica Cotrone told Biopharma-Reporter.com.

“No new additional studies were requested and Shire promptly addressed the FDA’s comments,”​ she added, and the facility has now been given the go-ahead to manufacture the drug for the US market, following the site’s European approval in February 2012.

Speaking last week in a conference call to discuss end of year results (transcript here​), CEO Flemming Ornskov said the decision meant Shire was now “unencumbered in [its] supply for VPRIV,”​ and would complement the 12% growth in sales the orphan disease drug had seen over the last twelve months.

Single-Use Manufacture

“VPRIV is made in [4 x 2,000L] single-use bioreactor and also uses disposable technology,”​ Cotrone told us.

In fact, the entire Lexington plant uses only single-use and disposable technology in its biopharmaceutical production and, according to the firm, is the first commercially licensed facility in the world to do so.

“I believe there are others that have taken our lead, but we were the first,”​ Cotrone said, adding she was unaware of any competitor that had caught up with Shire in the move to single-use.

“There are really no downsides to running the plant dependent on single-use technology​,” she continued. “In addition to increasing capacity and reducing manufacturing risk, utilization of single-use technology requires approximately 80% less water and 50% less energy than a conventional manufacturing plant.”

Gaucher Disease

The disease is caused by mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase, causing an accumulation of glucocerebroside which can lead to organomegaly, anemia and thrombocytopenia.

Approval of VPRIV was pushed through in 2010 after manufacturing issues caused a shortage​ of Sanofi subsidiary Genzyme’s gaucher disease drug Cerezyme.

The disease only affects around 5,000 patients worldwide but Cerezyme is sold at an annual cost of around $200,000, making it one of the most expensive drugs on the market, and 2012 sales clocked in at $870m, according to Sanofi’s 2012 results​.

As well as Cerezyme, VPRIV competes with Actelion’s Zavesca and Protalix’s Elelyso, which is marketed by Pfizer outside Israel.

Furthermore, according to a Shire report​, the firm is “aware of four companies that have early stage biosimilar development programs for Gaucher disease: JCR; Isu-Abxis; Harvest Moon; and Dong A Pharmaceuticals.”

Correction - the original article stated the drug was made in an 8,000L single-use bioreactor

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