ELELYSO (Page 4 of 4)

14.2 Clinical Trial in Patients Switching from Imiglucerase Treatment to ELELYSO

The safety and efficacy of ELELYSO were assessed in 31 patients (26 adult and 5 pediatric patients) with Type 1 Gaucher disease who were switched from imiglucerase to ELELYSO. The trial was 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. Patients were required to be clinically stable and have a stable biweekly dose of imiglucerase for at least 6 months prior to enrollment. Patients were 6 to 66 years of age (mean age 42 years, including pediatric patients), and 55% were male. Imiglucerase therapy was stopped, and treatment with ELELYSO was administered every other week at the same number of units as each patient’s previous imiglucerase dose. If needed, adjustment of dosage was allowed during the study in order to maintain stability of clinical parameters (i.e., spleen volume, liver volume, platelet count, and hemoglobin).

Mean (SD) organ volumes and hematologic values remained stable through 9 months of ELELYSO treatment. At baseline, spleen volume was 5.2 (4.5) MN, liver volume was 1.0 (0.3) MN, platelet count was 161,137 (73,387)/mm3 , and hemoglobin was 13.5 (1.4) g/dL. After 9 months of ELELYSO treatment, spleen volume was 4.8 (4.6) MN, liver volume was 1.0 (0.2) MN, platelet count was 161,167 (80,820)/mm3 , and hemoglobin was 13.4 (1.5) g/dL. ELELYSO dose remained unchanged in 30 of 31 patients. One patient required a dose increase at Week 24 (from 9.5 units/kg to 19 units/kg) for a platelet count of 92,000/mm3 at Week 22, which subsequently increased to 170,000/mm3 at Month 9.

Eighteen of the 26 adult patients who completed the 9-month clinical trial continued treatment with ELELYSO in an open-label extension trial for additional 27 months (total treatment 36 months). Patients maintained stability in clinical parameters (spleen volume, liver volume, platelet count and hemoglobin); however only 10 of 18 adult patients completed 27 months of ELELYSO treatment in the extension trial and only 7 patients had their spleen and liver volumes assessed at 36 months.

Five pediatric patients in the 9-month clinical trial who continued open-label treatment with ELELYSO for additional 24 months demonstrated stability in these clinical parameters.

16 HOW SUPPLIED/STORAGE AND HANDLING

ELELYSO (taliglucerase alfa) for injection is supplied as a sterile, preservative-free, white to off-white lyophilized powder in a single-dose vial. Each vial of ELELYSO contains 200 units of taliglucerase alfa.

Each carton contains one vial (NDC 0069-0106-01).

Store ELELYSO refrigerated at 2° C to 8° C (36° F to 46° F) in the original carton to protect from light. Do not freeze.

17 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 [see Warnings and Precautions (5.1)].

Manufactured by
Pfizer Inc.
NY, NY 10017
US License No. 2001

Logo

LAB-0610-11.0

PRINCIPAL DISPLAY PANEL — 200 Unit Vial Label

NDC 0069-0106-01

Pfizer

Elelyso®
(taliglucerase alfa)
for injection

200 units/vial

For intravenous infusion only

Single-Dose Vial. Discard
any unused portion. Rx only

PRINCIPAL DISPLAY PANEL -- 200 Unit Vial Label
(click image for full-size original)

PRINCIPAL DISPLAY PANEL — 200 Unit Vial Carton

NDC 0069-0106-01

Pfizer

Elelyso®
(taliglucerase alfa)
for injection

200 units/vial

For intravenous infusion only

Single-Dose Vial. Discard any
unused portion. Rx only

PRINCIPAL DISPLAY PANEL -- 200 Unit Vial Carton
(click image for full-size original)
ELELYSO taliglucerase alfa injection, powder, lyophilized, for solution
Product Information
Product Type HUMAN PRESCRIPTION DRUG Item Code (Source) NDC:0069-0106
Route of Administration INTRAVENOUS DEA Schedule
Active Ingredient/Active Moiety
Ingredient Name Basis of Strength Strength
TALIGLUCERASE ALFA (TALIGLUCERASE ALFA) TALIGLUCERASE ALFA 200 U in 5 mL
Inactive Ingredients
Ingredient Name Strength
MANNITOL 206.7 mg in 5 mL
POLYSORBATE 80 0.56 mg in 5 mL
SODIUM CITRATE, UNSPECIFIED FORM 30.4 mg in 5 mL
Packaging
# Item Code Package Description Multilevel Packaging
1 NDC:0069-0106-01 1 VIAL, GLASS in 1 CARTON contains a VIAL, GLASS
1 5 mL in 1 VIAL, GLASS This package is contained within the CARTON (0069-0106-01)
Marketing Information
Marketing Category Application Number or Monograph Citation Marketing Start Date Marketing End Date
BLA BLA022458 05/01/2012
Labeler — Pfizer Laboratories Div Pfizer Inc (134489525)
Registrant — Pfizer Inc (113480771)
Establishment
Name Address ID/FEI Operations
Pharmacia and Upjohn Company LLC 618054084 LABEL (0069-0106), MANUFACTURE (0069-0106), PACK (0069-0106)
Establishment
Name Address ID/FEI Operations
Pfizer Ireland Pharmaceuticals 985586408 ANALYSIS (0069-0106)

Revised: 07/2021 Pfizer Laboratories Div Pfizer Inc

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