DOCSLIB.ORG

761090Orig1s000

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
761090Orig1s000 CENTER FOR DRUG EVALUATION AND RESEARCH APPLICATION NUMBER: 761090Orig1s000 MULTI-DISCIPLINE REVIEW Summary Review Office Director Cross Discipline Team Leader Review Clinical Review Non-Clinical Review Statistical Review Clinical Pharmacology Review BLA 761090 Multi-disciplinary Review and Evaluation Takhzyro (lanadelumab) NDA/BLA Multi-disciplinary Review and Evaluation Application Type BLA Application Number(s) 761090 Priority or Standard Priority Submit Date(s) December 26, 2017 Received Date(s) December 26, 2017 PDUFA Goal Date August 24, 2018 Division/Office DPARP/ODE II Review Completion Date August 22, 2018 Established Name Lanadelumab (Proposed) Trade Name Takhzyro Pharmacologic Class Biologic Code name SHP643, DX-2930 Applicant Dyax Formulation(s) Ready to use solution for injection Dosing Regimen 300 mg every 2 weeks (b) (4) Applicant Proposed For prophylaxis to prevent attacks of Indication(s)/Population(s) hereditary angioedema (HAE) in patients 12 years and older. Recommendation on Approval Regulatory Action Recommended For prophylaxis to prevent attacks of hereditary angioedema Indication(s)/Population(s) (HAE) in patients 12 years and older (if applicable) 1 Version date: September 8, 2017 for initial rollout (NME/original BLA reviews) Reference ID: 4311123 BLA 761090 Multi-disciplinary Review and Evaluation Takhzyro (lanadelumab) Table of Contents Reviewers of Multi-Disciplinary Review and Evaluation ................................................................ 9 Additional Reviewers of Application ............................................................................................... 9 Glossary ......................................................................................................................................... 10 1 Executive Summary ............................................................................................................... 13 Product Introduction ...................................................................................................... 13 Conclusions on the Substantial Evidence of Effectiveness ............................................ 13 Benefit-Risk Assessment ................................................................................................ 13 Patient Experience Data ................................................................................................. 16 2 Therapeutic Context .............................................................................................................. 18 Analysis of Condition ...................................................................................................... 18 Analysis of Current Treatment Options ......................................................................... 20 3 Regulatory Background ......................................................................................................... 21 U.S. Regulatory Actions and Marketing History ............................................................. 21 Summary of Presubmission/Submission Regulatory Activity ........................................ 21 4 Significant Issues from Other Review Disciplines Pertinent to Clinical Conclusions on Efficacy and Safety................................................................................................................. 23 Office of Scientific Investigations (OSI) .......................................................................... 23 Product Quality .............................................................................................................. 23 Clinical Microbiology ...................................................................................................... 23 Devices and Companion Diagnostic Issues .................................................................... 24 5 Nonclinical Pharmacology/Toxicology................................................................................... 24 Executive Summary ........................................................................................................ 24 Referenced NDAs, BLAs, DMFs ....................................................................................... 25 Pharmacology ................................................................................................................. 25 ADME/PK ........................................................................................................................ 30 Toxicology ....................................................................................................................... 32 General Toxicology .................................................................................................. 32 Genetic Toxicology .................................................................................................. 34 Carcinogenicity ........................................................................................................ 34 2 Version date: September 8, 2017 for initial rollout (NME/original BLA reviews) Reference ID: 4311123 BLA 761090 Multi-disciplinary Review and Evaluation Takhzyro (lanadelumab) Reproductive and Developmental Toxicology ........................................................ 37 5.6 Exposure margins ............................................................................................................. 49 5.7 Labeling .......................................................................................................................... 51 6 Clinical Pharmacology ............................................................................................................ 54 Executive Summary ........................................................................................................ 54 Summary of Clinical Pharmacology Assessment ............................................................ 56 Pharmacology and Clinical Pharmacokinetics ........................................................ 56 Pharmacokinetics in HAE Patients .................................................................................... 56 Pharmacokinetics in Special Populations ......................................................................... 56 Immunogenicity ................................................................................................................ 56 Pharmacodynamics ........................................................................................................... 57 General Dosing and Therapeutic Individualization ................................................. 57 Comprehensive Clinical Pharmacology Review ............................................................. 57 General Pharmacology and Pharmacokinetic Characteristics ................................ 57 Clinical Pharmacology Questions ............................................................................ 68 7 Sources of Clinical Data and Review Strategy ....................................................................... 81 Table of Clinical Studies .................................................................................................. 81 Review Strategy .............................................................................................................. 82 8 Clinical and Statistical Evaluation .......................................................................................... 82 Review of Relevant Individual Trials Used to Support Efficacy ...................................... 82 DX-2930-03 (HELP Study) ........................................................................................ 82 8.1.1.1. DX2930-03 Study Results ................................................................................. 91 DX-2930-04 (HELP Extension Study) ..................................................................... 110 8.1.2.1. DX2930-04 Study Results ............................................................................... 117 Assessment of Efficacy Across Trials ..................................................................... 119 Integrated Assessment of Effectiveness ............................................................... 120 Review of Safety ........................................................................................................... 121 Safety Review Approach ....................................................................................... 121 Review of the Safety Database ............................................................................. 121 Adequacy of Applicant’s Clinical Safety Assessments .......................................... 124 Safety Results ........................................................................................................ 124 3 Version date: September 8, 2017 for initial rollout (NME/original BLA reviews) Reference ID: 4311123 BLA 761090 Multi-disciplinary Review and Evaluation Takhzyro (lanadelumab) Analysis of Submission-Specific Safety Issues ....................................................... 129 Safety Analyses by Demographic Subgroups ........................................................ 130 Specific Safety Studies/Clinical Trials .................................................................... 130 Additional Safety Explorations .............................................................................. 136 Safety in the Postmarket Setting .......................................................................... 137 Integrated Assessment of Safety ..................................................................
Load more

Recommended publications
  • Pharmacy Prior Authorization Grid ALTCS, and Pharmacy
    Please Note: Refer to the other PA grids for applicable covered services that require PA. PA Grids: Medical, Behavioral Health, Pharmacy Prior Authorization Grid ALTCS, and Pharmacy. (Effective Date of Service 1/1/2021) Injectables that require Prior Authorization All chemotherapeutic drugs must be used for FDA-approved indications and/or in accordance with NCCN guidelines *Indicates prior authorization required if billed charges are greater than $400 PA Required HMO 13 HCPCS Short Description (BUCA- Code SNP) 90378 Respiratory Syncytial Virus Immune Globulin Yes C9036 Patisiran Yes C9047 Caplacizumab-yhdp Yes C9061 Teprotumumab-trbw Yes C9063 Eptinezumab-jjmr Yes C9131 Factor VIII antihemophilic factor pegylated-auci Yes C9132 Prothrombin Complex Concentrate (Human), Kcentra Yes C9133 Factor IX (Antihemophilic Factor, Recombinant), Rixibus Yes C9399 Mipomersen (Kynamro) Yes J0129 Abatacept Yes J0135 Adalimumab Yes J0178 Aflibercept Yes J0179 Brolucizumab-dbll, 1 mg Yes J0180 Agalsidase Beta Yes J0205 Alglucerase Yes J0215 Alefacept Yes J0220 Alglucosidase Alfa (Myozyme) Yes J0221 Alglucosidase Alfa (Lumizyme) Yes J0222 Patisiran, 0.1 mg Yes J0223 Givosiran 0.5 mg Yes J0256 Alpha 1-Proteinase Inhibitor Yes J0257 Alpha 1-Proteinase Inhibitor (Glassia) Yes J0275 Alprostadil Urethral Suppository Yes J0490 Belimumab Yes J0517 Benralizumab Yes J0567 Cerliponase alfa Yes J0570 Buprenorphine implant Yes J0584 Burosumab-twza 1 mg Yes J0585 Onabotulinumtoxina (Botox) Yes J0586 Abobotulinumtoxina (Dysport) Yes J0587 Rimabotulinumtoxinb (Myobloc)
    [Show full text]
  • Kalbitor® (Ecallantide)
    Kalbitor® (ecallantide) (Subcutaneous) Document Number: MODA-0167 Last Review Date: 10/01/2020 Date of Origin: 08/27/2013 Dates Reviewed: 04/2014, 09/2014, 03/2015, 06/2015, 09/2015, 12/2015, 03/2016, 06/2016, 09/2016, 12/2016, 03/2017, 06/2017, 09/2017, 12/2017, 03/2018, 06/2018 10/2018, 10/2019, 03/2020, 10/2020 I. Length of Authorization Coverage will be provided for 12 weeks and is eligible for renewal (unless otherwise specified). The cumulative amount of medication(s) the patient has on-hand, indicated for the acute treatment of HAE, will be taken into account when authorizing. The authorization will provide a sufficient quantity in order for the patient to have a cumulative amount of HAE medication on-hand in order to treat up to 4 acute attacks per 4 weeks for the duration of the authorization (unless otherwise specified). II. Dosing Limits A. Quantity Limit (max daily dose) [NDC unit]: Kalbitor 10 mg vial: 24 vials per 28 days B. Max Units (per dose and over time) [HCPCS Unit]: 240 billable units per 28 days III. Initial Approval Criteria 1 Coverage is provided in the following conditions: Patient is at least 12 years of age; AND Universal Criteria 1,13,18 Must be prescribed by, or in consultation with, a specialist in: allergy, immunology, hematology, pulmonology, or medical genetics; AND Confirmation the patient is avoiding the following possible triggers for HAE attacks: o Estrogen-containing oral contraceptive agents AND hormone replacement therapy; AND o Antihypertensive agents containing ACE inhibitors; AND o Dipeptidyl peptidase IV (DPP-IV) inhibitors (e.g., sitagliptin); AND o Neprilysin inhibitors (e.g., sacubitril) Moda Health Plan, Inc.
    [Show full text]
  • The Use of Radiotherapy in Hereditary Angioedema Type 1- C1 Inhibitor Deficiency
    Avances en Biomedicina ISSN: 2477-9369 ISSN: 2244-7881 [email protected] Universidad de los Andes Venezuela The use of radiotherapy in Hereditary Angioedema Type 1- C1 Inhibitor deficiency Lara de la Rosa, María del Pilar; Conde Alcañiz, Amparo; Moreno Ramírez, David; Illescas Vacas, Ana; Guardia Martínez, Pedro The use of radiotherapy in Hereditary Angioedema Type 1- C1 Inhibitor deficiency Avances en Biomedicina, vol. 7, no. 2, 2018 Universidad de los Andes, Venezuela Available in: https://www.redalyc.org/articulo.oa?id=331359393006 PDF generated from XML JATS4R by Redalyc Project academic non-profit, developed under the open access initiative Casos Clínicos e use of radiotherapy in Hereditary Angioedema Type 1- C1 Inhibitor deficiency Uso de radioterapia en Angioedema hereditario por déficit de C1 inhibidor tipo I María del Pilar Lara de la Rosa [email protected] University Hospital Virgen Macarena, España Amparo Conde Alcañiz University Hospital Virgen Macarena, España David Moreno Ramírez University Hospital Virgen Macarena, España Ana Illescas Vacas University Hospital Virgen Macarena, España Pedro Guardia Martínez University Hospital Virgen Macarena, España Avances en Biomedicina, vol. 7, no. 2, 2018 Universidad de los Andes, Venezuela Received: 27 February 2018 Accepted: 21 June 2018 Abstract: We present a clinical case of a 72 year old man with Hereditary Angioedema Type 1. It´s a rare, potentially fatal disease, especially due to causing episodes of Redalyc: https://www.redalyc.org/ laryngeal angioedema. He has a past medical history of lip squamous-cell skin cancer, articulo.oa?id=331359393006 which is currently relapsing, with lateral margins of the surgical resection affected requiring treatment with local radiotherapy.
    [Show full text]
  • Ehealth DSI [Ehdsi V2.2.2-OR] Ehealth DSI – Master Value Set
    MTC eHealth DSI [eHDSI v2.2.2-OR] eHealth DSI – Master Value Set Catalogue Responsible : eHDSI Solution Provider PublishDate : Wed Nov 08 16:16:10 CET 2017 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 1 of 490 MTC Table of Contents epSOSActiveIngredient 4 epSOSAdministrativeGender 148 epSOSAdverseEventType 149 epSOSAllergenNoDrugs 150 epSOSBloodGroup 155 epSOSBloodPressure 156 epSOSCodeNoMedication 157 epSOSCodeProb 158 epSOSConfidentiality 159 epSOSCountry 160 epSOSDisplayLabel 167 epSOSDocumentCode 170 epSOSDoseForm 171 epSOSHealthcareProfessionalRoles 184 epSOSIllnessesandDisorders 186 epSOSLanguage 448 epSOSMedicalDevices 458 epSOSNullFavor 461 epSOSPackage 462 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 2 of 490 MTC epSOSPersonalRelationship 464 epSOSPregnancyInformation 466 epSOSProcedures 467 epSOSReactionAllergy 470 epSOSResolutionOutcome 472 epSOSRoleClass 473 epSOSRouteofAdministration 474 epSOSSections 477 epSOSSeverity 478 epSOSSocialHistory 479 epSOSStatusCode 480 epSOSSubstitutionCode 481 epSOSTelecomAddress 482 epSOSTimingEvent 483 epSOSUnits 484 epSOSUnknownInformation 487 epSOSVaccine 488 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 3 of 490 MTC epSOSActiveIngredient epSOSActiveIngredient Value Set ID 1.3.6.1.4.1.12559.11.10.1.3.1.42.24 TRANSLATIONS Code System ID Code System Version Concept Code Description (FSN) 2.16.840.1.113883.6.73 2017-01 A ALIMENTARY TRACT AND METABOLISM 2.16.840.1.113883.6.73 2017-01
    [Show full text]
  • Kalbitor® (Ecallantide)
    Kalbitor® (ecallantide) (Subcutaneous) Document Number: IC-0167 Last Review Date: 03/03/2020 Date of Origin: 08/27/2013 Dates Reviewed: 04/2014, 09/2014, 03/2015, 06/2015, 09/2015, 12/2015, 03/2016, 06/2016, 09/2016, 12/2016, 03/2017, 06/2017, 09/2017, 12/2017, 03/2018, 06/2018 10/2018, 10/2019, 03/2020 I. Length of Authorization Coverage will be provided for 12 weeks and is eligible for renewal (unless otherwise specified). The cumulative amount of medication(s) the patient has on-hand, indicated for the acute treatment of HAE, will be taken into account when authorizing. The authorization will provide a sufficient quantity in order for the patient to have a cumulative amount of HAE medication on-hand in order to treat up to 4 acute attacks per 4 weeks for the duration of the authorization (unless otherwise specified). II. Dosing Limits A. Quantity Limit (max daily dose) [NDC unit]: − Kalbitor 10 mg vial: 24 vials per 28 days B. Max Units (per dose and over time) [HCPCS Unit]: • 240 billable units per 28 days 1-15 III. Initial Approval Criteria Coverage is provided in the following conditions: Universal Criteria: • Must be prescribed by, or in consultation with, a specialist in: allergy, immunology, hematology, pulmonology, or medical genetics; AND • Confirmation the patient is avoiding the following possible triggers for HAE attacks: o Estrogen-containing oral contraceptive agents AND hormone replacement therapy; AND o Antihypertensive agents containing ACE inhibitors; AND Treatment of acute attacks of Hereditary Angioedema (HAE) † • Patient must be at least 12 years of age; AND Proprietary & Confidential © 2020 Magellan Health, Inc.
    [Show full text]
  • Ecallantide (Kalbitor) Reference Number: CP.PHAR.177 Effective Date: 03/16 Coding Implications Last Review Date: 03/17 Revision Log
    Clinical Policy: Ecallantide (Kalbitor) Reference Number: CP.PHAR.177 Effective Date: 03/16 Coding Implications Last Review Date: 03/17 Revision Log See Important Reminder at the end of this policy for important regulatory and legal information. Description The intent of the criteria is to ensure that patients follow selection elements established by Centene® clinical policy for ecallantide (Kalbitor®). Policy/Criteria It is the policy of health plans affiliated with Centene Corporation® that Kalbitor is medically necessary when the following criteria are met: I. Initial Approval Criteria A. Hereditary Angioedema (HAE) (must meet all): 1. Diagnosis of HAE confirmed by one of the following (a or b): a. Low C4 level and low C1-INH antigenic or functional level (see Appendix B); b. Normal C4 level and normal C1-INH levels, and all of the following (i - iii): i. History of recurrent angioedema; ii. Family history of angioedema; iii. Other types of angioedema have been ruled out (e.g., ACE-I/ARB-associated or other drug-induced angioedema, allergic angioedema, nonhistaminergic angioedema); 2. Prescribed for treatment of acute attacks; 3. Prescribed dose of Kalbitor does not exceed 30 mg per dose (1 carton [3 vials] per dose) with up to 2 doses administered in a 24 hour period. Approval duration: 12 months (no more than 4 doses per month) B. Other diagnoses/indications: Refer to CP.PHAR.57 - Global Biopharm Policy. II. Continued Approval A. Hereditary Angioedema (must meet all): 1. Currently receiving medication via Centene benefit or member has previously met initial approval criteria; 2. Documentation of positive response to therapy; 3.
    [Show full text]
  • References I-122 1. Kalbitor (Ecallantide) Injection, For
    References I-122 1. Kalbitor (ecallantide) injection, for subcutaneous use [package insert]. Dyax Corp. Burlington, MA. Revised 03/2015. 2. Clinical Pharmacology Compendia [database online]. Tampa FL: Gold Standard, Inc. Ecallantide. 2020. 3. Micromedex®Solutions Compendia. 2020. Ecallantide. 4. Ecallantide In: AHFS Drug Information Online Electronic Medical Library. Bethesda, MD: American Society of Health-System Pharmacists. 2020. 5. Firazyr (icatibant) injection, for subcutaneous use [package insert]. Takeda Pharmaceuticals America, Inc. Lexington, MA. Revised 08/2020. 6. Ruconest (C1 Esterase inhibitor, recombinant) for intravenous use [package insert]. Pharming Healthcare Inc. Bridgewater, NJ. Revised 12/2019. 7. Berinert (C1 Esterase inhibitor, human) for intravenous use [package insert]. CSL Behring LLC. Kankakee, IL. Revised 04/2019. 8. Cinryze (C1 esterase inhibitor, human) for intravenous use [package insert]. Shire ViroPharma Inc. Lexington, MA. Revised 06/2018. 9. Henao M, Kraschnewski J, Kelbel T, et Diagnosis and screening of patients with hereditary angioedema in primary care. Ther Clin Risk Manag. 2016;12:701-711. 10. Bernstein J, Riedl M, Zacek L. Facilitating home-based treatment of hereditary angioedema. Allergy Asthma Proc. 2015;36:92-99. 11. S. Hereditary Angioedema Association. (haea). US hereditary angioedema association medical advisory board 2013 recommendations for the management of hereditary angioedema due to c1 inhibitor deficiency. Accessed April 13, 2017. 12. Henry, H, Kaliner M. Angioedema Treatment & Management. Medscape. Last updated April 12, Histaminergic Angioedema (IH-AAE). 13. Bernstein JA, Riedl M, Zacek Facilitating home-based treatment of hereditary angioedema. Allergy Asthma Proc. 2015;92-99. 14. Polinski JM, Kowal MK, Gagnon M, et Home infusion: safe clinically effective, patient preferred, and cost saving.
    [Show full text]
  • Place of Service for Medical Infusions “Notification” POLICY EFFECTIVE OCTOBER 1, 2021
    Corporate Medical Policy: Place of Service for Medical Infusions “Notification” POLICY EFFECTIVE OCTOBER 1, 2021 Related Corporate Medical Policies with Applicable Restricted Product(s): • Abatacept (Orencia®) • Alpha 1-Antitrypsin Inhibitor Therapy • Belimumab (Benlysta®) • Burosumab-twza (Crysvita®) • Canakinumab (Ilaris®) • Certolizumab pegol (Cimzia®) • Crizanlizumab-tmca (Adakveo®) • Eculizumab (Soliris®) • Edaravone (Radicava®) • Enzyme Replacement Therapy (ERT) for Lysosomal Storage Disorders • Eptinezumab-jjmr (Vyepti™) • Evinacumab-dgnb (Evkeeza™) • Fosdenopterin (Nulibry™) • Givosiran (Givlaari®) • Golimumab (Simponi Aria®) • Guselkumab (Tremfya®) • Ibalizumab-uiyk (Trogarzo®) • Immunoglobulin Therapy • Inebilizumab-cdon (Uplizna™) • Infliximab (Remicade®) and Infliximab Biosimilars • Interleukin-5 Antagonists • Letermovir (Prevymis™) • Lumasiran (Oxlumo™) • Luspatercept-aamt (Reblozyl®) • Natalizumab (Tysabri®) • Ocrelizumab (Ocrevus®) • Omalizumab (Xolair®) BLUE CROSS®, BLUE SHIELD® and the Cross and Shield Symbols are registered marks of the Blue Cross and Blue Shield Association, an association of independent Blue Cross and Blue Shield Plans. Blue Cross NC is an independent licensee of the Blue Cross and Blue Shield Association. All other marks are the property of their respective owners. Page 1 October 2021 • Patisiran (Onpattro®) • Pegcetacoplan (Empaveli™) • Plasminogen, human-tvmh (Ryplazim®) • Ravulizumab-cwvz (Ultomiris®) • Romiplostim (NPlate®) • Romosozumab-aqqg (Evenity™) • Somatostatin Analogs • Teprotumumab-trbw
    [Show full text]
  • Download Guide
    Considerations for starting a preventative therapy A guide to understanding the impact of your hereditary angioedema (HAE) and ways to manage it To help guide your discussion with your healthcare provider, answer the following questions. Together, with this information, you and your healthcare provider can discuss where you are in your journey, your satisfaction with your current treatment plan, and if ORLADEYOTM (berotralstat) may be right for you. Living with HAE In the last 6 months • How many attacks have you experienced? ______ On a scale of mild to severe, rate the average severity of your attacks, keeping in mind where on the body attacks occurred. Mild Moderate Severe 1 2 3 4 5 • What triggers your HAE attacks? (check all that apply) Stress​ Trauma Illness Other • How many days of work/school/social events have been disrupted because of your HAE? 0-5 6-10 11-15 More than 15 On a scale of not at all to all the time, how often do you think about your HAE? Not at all Sometimes All the time 1 2 3 4 5 What is your HAE action plan? WHAT IS ORLADEYOTM (berotralstat)? ORLADEYO (or-luh-DAY-oh) is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in adults and children 12 years of age and older. It is not known if ORLADEYO is safe and effective in children under 12 years of age. It is not known if ORLADEYO is safe and effective to treat an acute HAE attack, therefore ORLADEYO should not be used to treat an acute HAE attack.
    [Show full text]
  • (PDF) Download
    www.scottishmedicines.org.uk SMC2206 lanadelumab 300mg solution for injection (Takhzyro®) Shire, part of Takeda UK Ltd 8 November 2019 The Scottish Medicines Consortium (SMC) has completed its assessment of the above product and advises NHS Boards and Area Drug and Therapeutic Committees (ADTCs) on its use in NHSScotland. The advice is summarised as follows: ADVICE: following a full submission assessed under orphan equivalent process lanadelumab (Takhzyro®) is accepted for restricted use within NHSScotland. Indication under review: For the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. SMC restriction: patients with HAE type I or II, who would otherwise be considered for long- term prophylaxis treatment with C1-esterase inhibitor. In a phase III study in patients with HAE, lanadelumab reduced the rate of angioedema attacks compared with placebo. This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a list price that is equivalent or lower. This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting. Chairman Scottish Medicines Consortium Published 09 December 2019 1 Indication For routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.1 Dosing Information The recommended starting dose of lanadelumab is 300mg by subcutaneous injection every 2 weeks. In patients who are stably attack free on treatment, a dose reduction of 300mg lanadelumab every 4 weeks may be considered, especially in patients with low weight. Lanadelumab is not intended for treatment of acute HAE attacks.
    [Show full text]
  • PM106 Ecallantide (Kalbitor)
    Department: Utilization Management Original Approval: 10/11/2013 Policy #: PM106 Last Approval: 01/07/2021 Title: Ecallantide (Kalbitor®) Approved By: UM Pharmacy Subcommittee Line(s) of Business WAH-IMC (HCA) BHSO Medicare Advantage (CMS) Medicare SNP (CMS) Cascade Select Documentation required to determine medical necessity for Ecallantide (Kalbitor®) History and/or physical examination notes and relevant specialty consultation notes that address the problem and need for the service: -Age -Diagnosis - Medication list -Prescribed by or in consultation with an immunologist or a physician that specializes in the treatment of HAE - BACKGROUND OVERVIEW Kalbitor, a plasma kallikrein inhibitor, is indicated for the treatment of acute attacks of hereditary angioedema (HAE) in patients ≥ 12 years of age.1 Potentially serious hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with Kalbitor. Kalbitor should only be administered by a healthcare professional with appropriate medical support to manage anaphylaxis and HAE. Disease Overview HAE due to C1 esterase inhibitor (C1-INH) deficiency has two subtypes: HAE type I and HAE type II. HAE diagnosis can be confirmed by measuring functional C1-INH protein levels (usually < 50% of normal in patients with HAE), C4 levels, and C1-INH antigenic levels.2,3 Patients with HAE type I have low C4 and C1-INH antigenic protein levels, along with low levels of functional C1-INH protein. Patients with HAE type II have low C4 and functional C1-INH protein level, with a normal or
    [Show full text]
  • John Hopkins Medicine Medicare Advantage Novologix Prior
    Advantage MD Medical Benefit (Part B) Drug Prior Authorization List *These prior authorization requirements impact all Advantage MD members.* Drug Name Please note: Brand name examples are HCPCS HCPCS Description included for reference only, and are not all- inclusive Q2053 Brexucabtagene autoleucel, up to 200 million autologous anti-cd19 car positive viable t Tecartus cells, including leukapheresis and dose preparation procedures, per therapeutic dose J0129 Injection, abatacept, 10 mg (code may be used for medicare when drug administered Orencia under the direct supervision of a physician, not for use when drug is self administered) J0178 Injection, aflibercept, 1 mg Eylea J0179 Injection, brolucizumab-dbll, 1 mg Beovu J0202 Injection, alemtuzumab, 1 mg Lemtrada J0221 Injection, alglucosidase alfa, (lumizyme), 10 mg Lumizyme J0222 Injection, patisiran, 0.1 mg Onpattro J0223 Injection, givosiran, 0.5 mg Givlaari J0256 Injection, alpha 1 proteinase inhibitor (human), not otherwise specified, 10 mg Aralast NP, Prolastin-C, Zemaira J0257 Injection, alpha 1 proteinase inhibitor (human), (glassia), 10 mg Glassia J0490 Injection, belimumab, 10 mg Benlysta J0517 Injection, benralizumab, 1 mg Fasenra J0584 Injection, burosumab-twza 1 mg Crysvita J0585 Injection, onabotulinumtoxina, 1 unit Botox J0586 Injection, abobotulinumtoxina, 5 units Dysport J0587 Injection, rimabotulinumtoxinb, 100 units Myobloc J0588 Injection, incobotulinumtoxin a, 1 unit Xeomin J0593 Injection, lanadelumab-flyo, 1 mg (code may be used for Medicare when drug Takhzyro
    [Show full text]