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FDA Approves Pfizer's Gaucher Drug for Children

AUGUST 28, 2014
James Radke

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

“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.”

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 fourteen 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).

According to the drug manufacturers,  the recommended dosage of Elelyso for treatment-naïve adult and pediatric patients four years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion.  Patients previously treated on a stable dosage of imiglucerase are recommended to begin treatment with Elelyso at that same dosage when they switch from imiglucerase to Elelyso. Dosage adjustments can be made based on achievement and maintenance of each patient’s therapeutic goals.

“It is important that children with this disease have access to a range of FDA-approved treatment options that are effective.”

The addition of the pediatric indication is welcome news.   Paige Kaplan, MB, BCh, Section of Biochemical Genetics (Metabolic Diseases), Children's Hospital of Philadelphia  stated “While Type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood,” adding,  “It is important that children with this disease have access to a range of FDA-approved treatment options that are effective.”

About Gaucher Disease

Gaucher disease is an inherited lysosomal storage disorder in which a deficiency of the enzyme glucocerebrosidase leads to the accumulation of the lipid glucocerebroside within the lysosomes of the monocyte-macrophage system. The most common form of this rare disease is Gaucher type 1 and its current treatment options are:
  • Cerezyme (imiglucerase for injection):  Cerezyme is the gold standard for ERT and has been treating people with Gaucher disease since 1994. It is administered by intravenous infusion every 2 weeks
  • Elelyso (taliglucerase alfa): Elelyso was approved by the FDA in June 2012 for the treatment of Gaucher disease type 1 in adults and approved August 2014 for treatment in children.  The key biochemical difference of Elelyso compared to the above enzyme replacement therapies is that it is derived from plant (carrot). It is administered by intravenous infusion every 2 weeks.
  • Vpriv (velaglucerase alfa for injection): Vpriv was approved in March 2010 of Vpriv and has also been shown to relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease.  It is also an enzyme replacement therapy and administered by intravenous infusion every 2 weeks.
  • Zavesca (miglustat). In addition to the above ERTs, there is also the option of using substrate reduction therapy for adult patients who cannot take enzyme replacement therapy. Zavesca® was approved by the FDA in July 2003. It is an oral medication (3 times per day).
  • Cerdelga (eliglustat): Cerdlelga is the first non ERT that can be used instead of ERT (unlike Zavesca that is only approved for patients who cannot take ERT). It was approved in August 2014.
 
 

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