ELELYSO (taliglucerase alfa) for injectionStatement of Medical Necessity & Physician Order/Prescription Phone 1-855-ELELYSO (1-855-353-5976) Fax 1-866-758-7135Patient name (last, first) ________________________________________________________ DOB Gender: M FAddress _____________________________________________________ City __________________________________State_______ ZIP _____________Home phone ____________________________________ Work/cell phone ___________________________________ SS # _____________________________Description and ICD-9 and/or ICD-10 (include all that apply)ICD-9/272.7 ICD-10/E75.22 Other __________________________________________________________________________________________Method of diagnosis _____________________________________________ Date of diagnosis ____________________________________________Height _____ feet _____ inches Weight ______ lb kg (circle one)Allergies ___________________________________________________________________Hgb/Hct levels ______________________________________ Date _________________Platelets ___________________________________________ Date _________________Splenectomy: Complete Partial No Date ______________________________MRI: Yes No Date __________________Spleen volume ___________________ cc Normal: Yes No Times normal _________________Liver volume ____________________ cc Normal: Yes No Times normal _________________Other _________________________________________________________________________________________________________________Start date ___________________ Dosage change: Yes NoELELYSO (taliglucerase alfa) for injection__________ U/kg NDC 0069 0106 01Skeletal Hx:Avascular necrosisBone crisisBone painErlenmeyer flaskdeformityInfarction(s)Refills ___________________ Frequency _____________________Provide any medical supplies, including syringes and needles, as necessary to safely administer prescribed medicationsOther orders __________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________I certify that the therapy described above is medically necessary and that the information provided above is accurate to the best of my knowledge.I also attest that I have obtained the patient’s affirmative authorization to release the above information and such other personal information asmay be necessary to any other healthcare provider participating in their care/services. If the patient is 18 years of age or younger I attest that Ihave obtained permission from the patient’s legal guardian.Physician InformationName ______________________________________________________________________ Fax ______________________________________________Address _____________________________________________________________________ NPI # ____________________________________________Phone ______________________________________________________________________ DEA # ___________________________________________Physician signature ______________________________________ _________________________________________ Date ___________________Substitution permittedDispense as writtenNPE00870/TAL460203-01 © 2012 Pfizer Inc. All rights reserved Printed in USA/May 2012Joint replacementsLytic lesion(s)Marrow infiltrationOsteopeniaPathological Fx(s)NoneRD 5-01nDosing & Administration1. Dosing of ELELYSO (taliglucerase alfa) for injection 1■ The recommended dose is 60 Units/kg of body weight administered onceevery 2 weeks as a 60-120 minute intravenous infusion– Patients currently being treated with imiglucerase for Type 1Gaucher disease can be switched to ELELYSO– Patients previously treated on a stable dose of imiglucerase arerecommended to begin treatment with ELELYSO at that same dosewhen they switch from imiglucerase to ELELYSO■ Dosage adjustments can be made based on achievement and maintenance ofeach patient’s therapeutic goals2. Calculate number of vials used 1■ ELELYSO is supplied in a 200-Unit vial of sterile, nonpyrogenic, lyophilizedproduct for intravenous infusion. The number of vials needed for eachinfusion can be calculated as follows:Patient’s weight (kg)60 Units per kg200 (Units per vial)x÷=Number of vials(Round up to the next whole vial)– Vials of ELELYSO should be stored at 2ºC to 8ºC (36ºF to 46ºF).Keep out of light. Do not use after the expiration date on the vialTo start your patients on ELELYSO (taliglucerase alfa) for injection:Physician Certification Order/Prescription Medical Information Diagnosis Patient InformationSee Statement of Medical Necessity form on following pages.Or visit ELELYSOHCP.com to get more informationand download forms.Please see Important Safety Information on page 1 and full Prescribing Information on following pages.
Coding & BillingProviders are responsible for the selection of appropriate codes. Listed beloware possible coding options, which may vary by health insurance or health careprovider. Pfizer Inc cannot guarantee that the billing codes listed below will result incoverage or payment. Please verify all codes with plan sponsors prior to submittingclaims. Since final coding is at the discretion of the health plan or health careprovider, the codes below should be used for reference purposes only.If you have questions or require more information about ELELYSO (taliglucerasealfa) for injection, please call Gaucher Personal Support (GPS) toll free at1-855-ELELYSO (1-855-353-5976) or visit ELELYSOHCP.com.Billing CodesNDC 0069 0106 01 ELELYSO (taliglucerase alfa) for injection 200 Units per vialICD-9-CM 272.7 Lipidosis (Gaucher disease) 2330.2Cerebral degeneration in generalized lipidoses (code firstunderlying disease as Gaucher disease 272.7) 2ICD-10-CM E75.22 Gaucher disease 3CPT 96365Intravenous infusion for therapy, prophylaxis, or diagnosis(specify substance or drug); initial, up to 1 hour 496366 Each additional hour 4HCPCS J3490 Unclassified drugs 5J3590 Unclassified biologics 5C9399 Unclassified drugs or biologics 5S9357Home infusion therapy, enzyme replacement intravenoustherapy; administrative services, professional pharmacyservices, care coordination, and all necessary suppliesand equipment 5Revenue 0258 IV solutions 60260 IV therapy, general 60261 Infusion pump therapy 60636 Drugs requiring detailed coding 6References: 1. Elelyso [package insert]. New York, NY: Pfizer Inc; 2012. 2. Centers for Disease Control.ICD-9-CM codes. Diseases and injuries tabular index. icd9cm.chrisendres.com/index.php?action=contents.Accessed November 7, 2011. 3. Centers for Disease Control. ICD-10-CM tabular list of diseases and injuries.ftp://ftp.cdc.gov/pub/Health_Statistics/NCHS/Publications/ICD10CM/2011/. Accessed November 8, 2011.4. American Medical Association. Current procedural terminology CPT® 2012 Professional Edition. 2011.5. HCPCS 2011 codes. ICD9Data.com. http://www.icd9data.com/HCPCS/2011/C/C9399.htm. AccessedDecember 2, 2011. 6. State of Oregon, Department of Consumer and Business Services. [Oregon.gov Website.] Expanded list of valid revenue codes. http://cbs.state.or.us/wcd/operations/edi/revenue_codes.pdf.Accessed April 2, 2012.Please see Important Safety Information on page 1 and full Prescribing Information on following pages.NPE00867A/TAL466400-01 © 2012 Pfizer Inc. All rights reserved. May 2012
8.4 Pediatric UseThe safety and effectiveness of ELELYSO in pediatric patients have not been established. One 8 year-old pediatric patient experienced a serious adversereaction (gastroenteritis).8.5 Geriatric UseDuring clinical studies 8 patients aged 65 or older were treated with ELELYSO. Clinical studies of ELELYSO did not include sufficient numbers of subjectsaged 65 and over to determine whether they respond differently from younger subjects.10 OVERDOSAGEThere is no experience with overdosage with ELELYSO.11 DESCRIPTIONTaliglucerase alfa, a hydrolytic lysosomal glucocerebroside-specific enzyme for intravenous infusion, is a recombinant active form of the lysosomal enzyme,β-glucocerebrosidase, which is expressed in genetically modified carrot plant root cells cultured in a disposable bioreactor system (ProCellEx®).β-Glucocerebrosidase (β-D-glucosyl-N-acylsphingosine glucohydrolase, E.C. 188.8.131.52) is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of theglycolipid glucocerebroside to glucose and ceramide.ELELYSO is produced by recombinant DNA technology using plant cell culture (carrot). Purified taliglucerase alfa is a monomeric glycoprotein containing 4 N-linked glycosylation sites (Mr = 60,800). Taliglucerase alfa differs from native human glucocerebrosidase by two amino acids at the N terminal and up to 7 aminoacids at the C terminal. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. Thesemannose-terminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells thataccumulate lipid in Gaucher disease.ELELYSO is supplied as a sterile, non-pyrogenic, lyophilized product. The quantitative composition of each 200 Unit vial is D-mannitol (206.7 mg), polysorbate80 (0.56 mg), sodium citrate (30.4 mg), and taliglucerase alfa (212 Units). Citric acid may be added to adjust the pH at the time of manufacture.A Unit is the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate para-nitrophenyl-β-D-glucopyranoside (pNP-Glc) perminute at 37°C. After reconstitution with Sterile Water for Injection the taliglucerase alfa concentration is 40 Units/mL [see Dosage and Administration (2)].Reconstituted solutions have a pH of approximately 6.0.12 CLINICAL PHARMACOLOGY12.1 Mechanism of ActionELELYSO catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. In clinical trials, ELELYSO reduced spleen and liver size, and improvedanemia and thrombocytopenia.12.3 PharmacokineticsIn Gaucher disease patients treated with 30 or 60 units/kg (N=29), pharmacokinetics were determined with the first dose and at 38 weeks.The pharmacokinetics of taliglucerase alfa appeared to be nonlinear with a greater than dose-proportional increase in exposure at the doses studied. Themedian systemic clearance (CL) values were approximately 30 L/hr and 20 L/hr for 30 and 60 units/kg, respectively. The median volume of distribution atsteady state (Vss) ranged from 7.30 to 11.7 L for both dose groups. At the end of infusion, taliglucerase alfa serum concentrations fell rapidly with a medianterminal half life of 18.9 to 28.7 minutes for both dose groups.No significant accumulation or change in taliglucerase alfa pharmacokinetics over time from Weeks 1 to 38 was observed with repeated doses of 30 or 60units/kg.Based on the limited data, there were no significant pharmacokinetic differences between male and female patients in this study.13 NONCLINICAL TOXICOLOGY13.1 Carcinogenesis, Mutagenesis, Impairment of FertilityLong-term studies in animals to evaluate carcinogenic potential or studies to evaluate mutagenic potential have not been performed with taliglucerase alfa.In a male and female fertility study in rats, taliglucerase alfa did not cause any significant adverse effect on male or female fertility parameters up to amaximum dose of 55 mg/kg/day (about 5 times the recommended human dose of 60 Units/kg based on the body surface area).14 CLINICAL STUDIES14.1 Study 1: Trial of ELELYSO as Initial TherapyThe safety and efficacy of ELELYSO was assessed in 31 adult patients with Type 1 Gaucher disease. The trial was a 9-month multi-center, double blind,randomized study in patients with Gaucher disease-related enlarged spleens (>8 times normal) and thrombocytopenia (
Change inSpleen Volume%BW Mean (SD)MN Mean (SD)-0.9 (0.4)-4.5 (2.1)-1.3 (1.1)-6.6 (5.4)Change inHemoglobing/dLMean (SD) 1.6 (1.4) 2.2 (1.4)Change in LiverVolume%BW Mean (SD)MN Mean (SD)-0.6 (0.5)-0.2 (0.2)-0.6 (0.4)-0.3 (0.2)Change inMean (SD) 11,427 (20,214) 41,494 (47,063)mm 3Platelet Count /Twenty-six previously treatment naïve patients continued to be treated with ELELYSO in an extension of this study (Study 3) in a blinded manner for a totaltreatment duration of 24 months. For the respective 30 and 60 Units/kg groups, mean (±SD) spleen volume (%BW) decreased -1.4 (±0.6) and -2.0 (±2.0);hemoglobin increased 1.3 (±1.7) g/dL and 2.4 (±2.3) g/dL; liver volume (%BW) decreased -1.1 (±0.5) and -1.0 (±0.7); and platelet count increased 28,433(±31,996) /mm3 and 72,029 (±68,157)/mm3.14.2 Study 2: Trial in Patients Switching from Imiglucerase to ELELYSOThe safety and efficacy of ELELYSO was assessed in 25patients with Type 1 Gaucher disease who were switched from imiglucerase to ELELYSO. Thetrial was a 9-month, multi-center, open-label, single arm study in patients who had been receiving treatment with imiglucerase at doses ranging from 11Units/kg to 60 Units/kg for a minimum of 2 years. Patients also were required to be clinically stable and to have a stable biweekly dose of imiglucerase forat least 6 months prior to enrollment. Patient age ranged from 13-66 years (mean age 45 years including pediatric) and 46% were male. Imiglucerasetherapy was stopped, and treatment with ELELYSO was administered every other week at the same number of units as each patient’s previous imiglucerasedose. Adjustment of dosage was allowed by study criteria if needed in order to maintain clinical parameters (i.e., hemoglobin, platelet count, spleen volume,and liver volume). One patient required a dose increase (from 9.5 Units/kg to 19 Units/kg at week 24) for a platelet count of 92,000/mm 3 at week 22, andresponded with a platelet count of 170,000mm 3 at month 9.Organ volumes and hematologic values remained stable on average through 9 months of ELELYSO treatment. At baseline, spleen volume %BW was 1.1% and MNwas 5.5; liver volume %BW was 2.4% and MN was 1.0; mean hemoglobin was 13.6 (± 1.57) g/dL; and mean platelet count was 160,447 (± 79,086) /mm3. At the ninemonth endpoint, spleen volume %BW was 1.0% and MN was 5.1; liver volume %BW was 2.3% and MN was 0.9; mean hemoglobin was 13.4 (± 1.6) g/dL and meanplatelet count was 165,654 (± 94,038) /mm3.16 HOW SUPPLIED/STORAGE AND HANDLINGELELYSO is available as a lyophilized powder, 200 Units per vial (NDC 0069 0106 01).Store ELELYSO at 2 to 8°C (36 to 46°F). Protect vials from light.17 PATIENT COUNSELING INFORMATION• Inform patients that ELELYSO is administered under the supervision of a healthcare professional as an intravenous infusion every other week. The infusiontypically takes 60 to 120 minutes.• Advise patients that ELELYSO may cause severe allergic reactions or infusion reactions. Patients should be counseled that they should be carefully reevaluatedfor treatment with ELELYSO if serious allergic reactions occur. Patients should also be counseled that infusion reactions can usually be managed byslowing the infusion rate, treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment withdecreased infusion rate. Patients should also be counseled that pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions [seeWarnings and Precautions (5.1, 5.2)].• Advise patients to report any adverse reactions while on ELELYSO treatment.Manufactured and distributed by:Pfizer LabsDivision of Pfizer Inc.New York, NY 10017Licensed from Protalix BiotherapeuticsLAB-0610-1.0Reference ID: 3124792