FDA Approves Pfizer's Gaucher Drug for Children

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Elelyso offers pediatric patients with Type 1 Gaucher disease another treatment option.

Elelyso offers pediatric patients with Type 1 Gaucher disease another treatment option.

Pfizer Inc. and Protalix BioTherapeutics, Inc. announced the US Food and Drug Administration (FDA) has approved Elelyso (taliglucerase alfa) for injection in pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.

In a press release, Pfizer’s Rory O’Connor, Senior Vice President, Global Medical Affairs, Global Innovative Pharma Business, stated, "The approval of Elelyso to treat pediatric patients with Type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease,” adding, “This pediatric indication, along with the recent announcement that Elelyso received kosher certification by the Orthodox Union (OU), reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”

The safety and efficacy of Elelyso were assessed in 14 pediatric patients with Type 1 Gaucher disease who were part of two clinical trials; a 12-month, multi-center, double-blind, randomized study in treatment-naïve patients that included children aged two to 13 years (n=9) and a 9-month, multi-center, open-label, single-arm study in patients who had been receiving treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of 2 years then switching to Elelyso. That study included children aged 6 to 16 years (n=5).

Click here to read the full article on Rare Disease Report.

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