Indication:
ELELYSO (taliglucerase alfa) for injection, for intravenous use
Initial US Approval: 2012
RECENT MAJOR CHANGES
Indications and Usage (1) 8/2014 Dosage and Administration,
Recommended Dosage (2.1) 8/2014
Dosage and Administration,
Preparation Instructions (2.2) 8/2014 Dosage and Administration,
Administration Instructions (2.3) 8/2014
Warnings and Precautions, Hypersensitivity Reactions
Including Anaphylaxis (5.1) 8/2014
Drug Name- Elelyso
Active Ingredient - Taliglucerase Alfa
For long- term enzyme replacement therapy to treat Gaucher disease,
a rare genetic disorder
Indication-
For long-term enzyme replacement therapy to treat Gaucher disease,
a rare genetic disorder
Approved by FDA on 1-5-2012 (Ref- FDA Approved List- 2012)
Adverse Reaction:
The most commonly reported adverse reactions (.5%) in clinical studies were pruritus,
flushing, headache, arthralgia, pain in extremity, abdominal pain, vomiting, fatigue,
back pain, dizziness, nausea and rash
Contra-Indications:
CONTRAINDICATIONS
None
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions Including Anaphylaxis:
Observe patients during and after the infusion; immediately discontinue infusion
if anaphylaxis occurs and initiate appropriate treatment.
Reduction in the infusion rate and/or pre-medication may prevent subsequent
reactions
Dosages/ Overdosage Etc:
Indication-
For long-term enzyme replacement therapy to treat Gaucher disease,
a rare genetic disorder
INDICATIONS AND USAGE
ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated
for long-term enzyme replacement therapy (ERT) for adult and pediatric
patients with a confirmed diagnosis of Type 1 Gaucher disease
DOSAGE AND ADMINISTRATION
Treatment-naïve adult and pediatric patients 4 years of age and older:
60 units/kg administered every other week as a 60 to 120 minute intravenous infusion.
Patients switching from imiglucerase:
Begin treatment with ELELYSO at the same unit/kg dose as the patient’s
previous imiglucerase dose. Physicians can make dosage adjustments based on
achievement and maintenance of each patient’s therapeutic goals
DOSAGE FORMS AND STRENGTHS
For injection: lyophilized powder for reconstitution with diluent, 200 unit single-use vials
Patient Information:
PATIENT COUNSELING INFORMATION
Hypersensitivity Reactions Including Anaphylaxis
Advise patients and caregivers that reactions related to administration and infusion
may occur during and after ELELYSO treatment, including life-threatening anaphylaxis
and severe hypersensitivity reactions.
Inform patients of the signs and symptoms of anaphylaxis and
hypersensitivity reactions, and have them seek medical care should signs
and symptoms occur
Inform patients that they should be carefully re-evaluated for treatment with ELELYSO
if serious hypersensitivity reactions, including anaphylaxis, occur.
Reduction of the infusion rate and/or pre-treatment with antihistamines, antipyretics
and/or corticosteroids may prevent subsequent reactions
Pharmacology/ Pharmacokinetics:
1. Mechanism of Action
Gaucher disease is an autosomal recessive disorder caused by mutations in the human
glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme
glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid
glucocerebroside into glucose and ceramide.
The enzymatic deficiency results in accumulation of substrate glucocerebroside
primarily in the lysosomal compartment of macrophages, giving rise to foam cells
or Gaucher cells, which accumulate in the liver, spleen and bone marrow.
2. Pharmacokinetics
Pharmacokinetics of taliglucerase alfa were evaluated in 38 patients
(29 adult and 9 pediatric patients) who received intravenous infusions of
ELELYSO 30 units/kg or 60 units/kg every other week.
ELELYSO 30 units/kg is not a recommended dose in treatment-naïve
Gaucher disease patients
.
Pregnancy and lactation:
USE IN SPECIFIC POPULATIONS
1. Pregnancy
Category B
Risk Summary
There are no adequate and well-controlled studies of ELELYSO in pregnant women.
In animal reproduction studies with taliglucerase alfa in pregnant rats at intravenous
doses up to 5 times the recommended human dose (RHD) and in pregnant rabbits
at intravenous doses up to 5 times the RHD , there was no evidence of harm to the fetus.
Because animal reproduction studies are not always predictive of human response,
ELELYSO should be used during pregnancy only if clearly needed.
2. Nursing Mothers
It is not known whether ELELYSO is present in human milk. Because many drugs are
present in human milk,caution should be exercised when ELELYSO is administered
to a nursing woman.
3. Pediatric Use
The use of ELELYSO for treatment of pediatric patients with Type 1 Gaucher disease
is supported by evidence of effectiveness from adequate and well-controlled trials
of ELELYSO in adults, with additional pharmacodynamic data from 5 pediatric patients
and pharmacokinetic data from 9 pediatric patients who participated in clinical trials
Data from 14 pediatric patients were included in the safety evaluation
There are insufficient data to inform dosing in patients less than 4 years of age.
Pediatric patients experienced a higher frequency of vomiting during ELELYSO
treatment (4 of 9 treatment-naïve patients) than adult patients, and this may be a
symptom of hypersensitivity reaction.
The frequencies of other adverse reactions were similar between pediatric and
adult patients
4. Geriatric Use
During clinical trials, 8 patients aged 65 or older were treated with ELELYSO.
Clinical trials of ELELYSO did not include sufficient numbers of patients aged 65
and over to determine whether they respond differently from younger patients
Manufacturer Details