DOCSLIB.ORG

Lysosomal Storage Disorders

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text

Load more

Lysosomal Storage Disorders Number: RX501.067 Effective Date: 01-15-2019 Coverage: *CAREFULLY CHECK STATE REGULATIONS AND/OR THE MEMBER CONTRACT* Medical policies are a set of written guidelines that support current standards of practice. They are based on current peer-reviewed scientific literature. A requested therapy must be proven effective for the relevant diagnosis or procedure. For drug therapy, the proposed dose, frequency and duration of therapy must be consistent with recommendations in at least one authoritative source. This medical policy is supported by FDA- approved labeling and nationally recognized authoritative references. These references include, but are not limited to: MCG care guidelines, DrugDex (IIb strength of recommendation or higher), NCCN Guidelines (IIb level of evidence or higher), NCCN Compendia (IIb level of evidence or higher), professional society guidelines, and CMS coverage policy. Enzyme-replacement therapy when utilized for the treatment of lysosomal storage disorders (LSD) may be considered medically necessary for the specific indications noted under each of the following enzyme- replacements therapies: I. Agalsidase beta (Fabrazyme®) Agalsidase beta (Fabrazyme) may be considered medically necessary for the use in members when the patient has a diagnosis of Fabry disease. Agalsidase beta (Fabrazyme) is considered experimental, investigational and/or unproven for the treatment of all other indications. II. Idursulfase (Elaprase™) Idursulfase (Elaprase™) may be considered medically necessary when: The patient is 5 years of age or greater; AND • The patient has a diagnosis of Hunter’s syndrome (mucopolysaccharidosis II [MPS II]). Idursulfase (Elaprase™) is considered experimental, investigational and/or unproven for the treatment of all other indications. III. Alglucosidase alfa (Myozyme®) Boeing_CS_PA_Lysosomal_Storage_Disorders_ProgSum_0121 Page 1 of 4 © Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 Alglucosidase alfa (Myozyme®) may be considered medically necessary for the treatment of infantile-onset Pompe disease (acid α-glucosidase [GAA] deficiency). Alglucosidase alfa (Myozyme®) is considered experimental, investigational and/or unproven for the treatment of all other indications, including but not limited to treatment of persons with other forms of Pompe disease. IV. Alglucosidase alfa (Lumizyme™) Alglucosidase alfa (Lumizyme™) may be considered medically necessary for patients with Pompe disease (GAA deficiency). Alglucosidase alfa (Lumizyme™) is considered experimental, investigational and/or unproven for the treatment of all other indications. V. Asfotase alfa (Strensiq™) Asfotase alfa (Strensiq™) may be considered medically necessary for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP). Asfotase alfa (Strensiq™) is considered experimental, investigational and/or unproven for all other indications. VI. Elosulfase alfa (Vimizim™) Elosulfase alfa (Vimizim™) may be considered medically necessaryforpatients 5 years of age or older with a documented clinical diagnosis of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). Elosulfase alfa (Vimizim™) is considered experimental, investigational and/or unproven for all other indications. VII. Naglazyme® (Galsulfase) Naglazyme® (Galsulfase) may be considered medically necessary for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). Naglazyme® (Galsulfase) is considered experimental, investigational and/or unproven for all other indications. VIII. Imiglucerase (Cerezyme®) Imiglucerase (Cerezyme®) may be considered medically necessary for long-term enzyme replacement therapy (ERT) for patients 2 years of age or greater with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions: Anemia; or Bone disease; or Boeing_CS_PA_Lysosomal_Storage_Disorders_ProgSum_0121 Page 2 of 4 © Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 Hepatomegaly or splenomegaly; or • Thrombocytopenia. Imiglucerase (Cerezyme®) is considered experimental, investigational and/or unproven for all other indications. IX. Laronidase (Aldurazyme®) Laronidase (Aldurazyme®) may be considered medically necessary for patients 6 months of age or greater with one of the following: Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I); or • Scheie form who have moderate to severe symptoms. Laronidase (Aldurazyme®) is considered experimental, investigational and/or unproven for all other indications, including but not limited to treatment of members with the Scheie form of MPS I who have mild symptoms. X. Sebelipase alfa (Kanuma™) Sebelipase alfa (Kanuma™) may be considered medically necessary when: • The patient is one month of age or greater; AND • The patient has a diagnosis of lysosomal acid lipase (LAL) deficiency (i.e. Wolman’s Disease). Sebelipase alfa (Kanuma™) is considered experimental, investigational and/or unproven for all other indications. XI. Taliglucerase alfa (Elelyso™) Taliglucerase alfa (Elelyso™) may be considered medically necessary for long-term ERT for patients 4 years of age or greater with a confirmed diagnosis of Type 1 Gaucher disease. Taliglucerase alfa (Elelyso™) is considered experimental, investigational and/or unproven for all other indications. XII. Velaglucerase alpha (VPRIV) Velaglucerase alpha (VPRIV) may be considered medically necessary for long-term ERT for patients 4 years of age or greater with type 1 Gaucher disease. Velaglucerase alpha (VPRIV) is considered experimental, investigational and/or unproven for all other indications. XIII. Vestronidase alfa-vjbk (MepseviiTM) Boeing_CS_PA_Lysosomal_Storage_Disorders_ProgSum_0121 Page 3 of 4 © Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 Vestronidase alfa-vjbk (MepseviiTM) may be considered medically necessary in patients 5 months of age or greater with a diagnosis of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Vestronidase alfa-vjbk (MepseviiTM) is considered experimental, investigational and/or unproven for all other indications. Length of Approval: 12 months TARGET AGENTS Agent GPI HCPCS Code Multisource Code Aldurazyme (laronidase) 2.9 mg / 5 mL injection 30906550002020 J1931 M, N, O, or Y Cerezyme (imiglucerase) 400 Unit injection 82700050002120 J1786 M, N, O, or Y Elaprase (idursulfase) 6 mg / 3 mL injection 30906850002020 J1743 M, N, O, or Y Elelyso (taliglucerase alfa) 200 unit injection 82700080102120 J3060 M, N, O, or Y Fabrazyme (agalsidase beta) 5 mg injection 30903610102110 J0180 M, N, O, or Y 35 mg injection 30903610102120 J0180 M, N, O, or Y Kanuma (sebelipase alfa) 20 mg / 10 mL injection 30906360002020 J2840 M, N, O, or Y Lumizyme (alglucosidase alfa) 50 mg injection 30907715002120 J0221 M, N, O, or Y Mepsevii (vestronidase alfa-vjbk) 10 mg / 5 mL injection 30907680202020 J3397 M, N, O, or Y Naglazyme (galsulfase) 1 mg / 1 mL injection 30907535002020 J1458 M, N, O, or Y Vimizim (elosulfase alfa) 5 mg / 5 mL injection 30907030052020 J1322 M, N, O, or Y Vpriv (velaglucerase alfa) 400 unit injection 82700085102120 J3385 M, N, O, or Y Boeing_CS_PA_Lysosomal_Storage_Disorders_ProgSum_0121 Page 4 of 4 © Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 .
Recommended publications
  • AHFS Pharmacologic-Therapeutic Classification System

    AHFS Pharmacologic-Therapeutic Classification System

    AHFS Pharmacologic-Therapeutic Classification System Abacavir 48:24 - Mucolytic Agents - 382638 8:18.08.20 - HIV Nucleoside and Nucleotide Reverse Acitretin 84:92 - Skin and Mucous Membrane Agents, Abaloparatide 68:24.08 - Parathyroid Agents - 317036 Aclidinium Abatacept 12:08.08 - Antimuscarinics/Antispasmodics - 313022 92:36 - Disease-modifying Antirheumatic Drugs - Acrivastine 92:20 - Immunomodulatory Agents - 306003 4:08 - Second Generation Antihistamines - 394040 Abciximab 48:04.08 - Second Generation Antihistamines - 394040 20:12.18 - Platelet-aggregation Inhibitors - 395014 Acyclovir Abemaciclib 8:18.32 - Nucleosides and Nucleotides - 381045 10:00 - Antineoplastic Agents - 317058 84:04.06 - Antivirals - 381036 Abiraterone Adalimumab; -adaz 10:00 - Antineoplastic Agents - 311027 92:36 - Disease-modifying Antirheumatic Drugs - AbobotulinumtoxinA 56:92 - GI Drugs, Miscellaneous - 302046 92:20 - Immunomodulatory Agents - 302046 92:92 - Other Miscellaneous Therapeutic Agents - 12:20.92 - Skeletal Muscle Relaxants, Miscellaneous - Adapalene 84:92 - Skin and Mucous Membrane Agents, Acalabrutinib 10:00 - Antineoplastic Agents - 317059 Adefovir Acamprosate 8:18.32 - Nucleosides and Nucleotides - 302036 28:92 - Central Nervous System Agents, Adenosine 24:04.04.24 - Class IV Antiarrhythmics - 304010 Acarbose Adenovirus Vaccine Live Oral 68:20.02 - alpha-Glucosidase Inhibitors - 396015 80:12 - Vaccines - 315016 Acebutolol Ado-Trastuzumab 24:24 - beta-Adrenergic Blocking Agents - 387003 10:00 - Antineoplastic Agents - 313041 12:16.08.08 - Selective
  • Ten Years of the Hunter Outcome Survey (HOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry Joseph Muenzer1, Simon A

    Ten Years of the Hunter Outcome Survey (HOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry Joseph Muenzer1, Simon A

    Muenzer et al. Orphanet Journal of Rare Diseases (2017) 12:82 DOI 10.1186/s13023-017-0635-z REVIEW Open Access Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry Joseph Muenzer1, Simon A. Jones2, Anna Tylki-Szymańska3, Paul Harmatz4, Nancy J. Mendelsohn5,6, Nathalie Guffon7, Roberto Giugliani8, Barbara K. Burton9, Maurizio Scarpa10,11, Michael Beck12, Yvonne Jangelind13, Elizabeth Hernberg-Stahl14, Maria Paabøl Larsen15,17, Tom Pulles16,18 and David A. H. Whiteman15* Abstract Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease- specific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered.
  • WO 2017/173059 Al 5 October 2017 (05.10.2017) P O P C T

    WO 2017/173059 Al 5 October 2017 (05.10.2017) P O P C T

    (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization I International Bureau (10) International Publication Number (43) International Publication Date WO 2017/173059 Al 5 October 2017 (05.10.2017) P O P C T (51) International Patent Classification: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, C12N 9/26 (2006.01) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (21) International Application Number: KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, PCT/US20 17/024981 MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (22) International Filing Date: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 30 March 2017 (30.03.2017) RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (25) Filing Language: English ZA, ZM, ZW. (26) Publication Language: English (84) Designated States (unless otherwise indicated, for every (30) Priority Data: kind of regional protection available): ARIPO (BW, GH, 62/3 15,400 30 March 2016 (30.03.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/457,584 10 February 2017 (10.02.2017) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 15/473,994 30 March 2017 (30.03.2017) US TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, (71) Applicant: AMICUS THERAPEUTICS, INC.
  • Publications in Scientific Journals (Peer Reviewed) 1

    Publications in Scientific Journals (Peer Reviewed) 1

    Last Updated July 2020 Publications in Scientific Journals (Peer Reviewed) 1. Eisengart JB, King KE, Shapiro EG, Whitley CB, Muenzer J. The nature and impact of neurobehavioral symptoms in neuronopathic Hunter syndrome. Mol Genet Metab Rep. 2019 Dec 20;22:100549. PMID: 32055445 2. Viskochil D, Clarke LA, Bay L, Keenan H, Muenzer J, Guffon N. Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry. Am J Med Genet A. 2019 Dec;179(12):2425-2432. PMID: 31639289 3. Clarke LA, Giugliani R, Guffon N, Jones SA, Keenan HA, Munoz-Rojas MV, Okuyama T, Viskochil D, Whitley CB, Wijburg FA, Muenzer J. Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry. Clin Genet. 2019 Clin Genet. 2019 Oct;96(4):281-289. PMID: 31194252 4. Taylor JL, Clinard K, Powell CM, Rehder C, Young SP, Bali D, Beckloff SE, Gehtland LM, Kemper AR, Lee S, Millington D, Patel HS, Shone SM, Woodell C, Zimmerman SJ, Bailey DB Jr, Muenzer J. The North Carolina Experience with Mucopolysaccharidosis Type I Newborn Screening. J Pediatr. 2019 Aug;211:193-200. PMID: 31133280 5. Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M; MPS Consensus Programme Steering Committee; MPS Consensus Programme Co-Chairs.
  • European Medicines Agency Accepts Marketing Authorization Application for Asfotase Alfa As a Treatment for Patients with Hypophosphatasia

    European Medicines Agency Accepts Marketing Authorization Application for Asfotase Alfa As a Treatment for Patients with Hypophosphatasia

    July 24, 2014 European Medicines Agency Accepts Marketing Authorization Application for Asfotase Alfa as a Treatment for Patients with Hypophosphatasia -- Application designated for review under accelerated assessment process -- CHESHIRE, Conn.--(BUSINESS WIRE)-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Marketing Authorization Application (MAA) for asfotase alfa, an investigational, first-in-class targeted enzyme replacement therapy for the treatment of hypophosphatasia (HPP), has been validated and granted accelerated assessment by the European Medicines Agency (EMA). The acceptance of this MAA marks the beginning of the review process in the European Union (EU) for this potential new treatment. "HPP is a devastating disease for patients and their families due to progressive deterioration of bones and muscle weakness, which can result in impaired respiratory function, severe disability and death," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "If approved, asfotase alfa would be the first therapy for patients with this life-threatening disorder." The EU filing includes positive data from 68 patients with pediatric-onset HPP (ranging from newborns to 66 years of age) enrolled in three pivotal prospective studies and their extensions, as well as a retrospective natural history study in infants. In April, Alexion initiated the rolling submission of a Biologics License Application (BLA) for asfotase alfa as a treatment for patients with HPP with the U.S. Food and Drug Administration (FDA). About
  • Enzyme Replacement Therapy Srx-0019 Policy Type ☒ Medical ☐ Administrative ☐ Payment

    Enzyme Replacement Therapy Srx-0019 Policy Type ☒ Medical ☐ Administrative ☐ Payment

    MEDICAL POLICY STATEMENT Original Effective Date Next Annual Review Date Last Review / Revision Date 06/15/2011 03/15/2017 10/04/2016 Policy Name Policy Number Enzyme Replacement Therapy SRx-0019 Policy Type ☒ Medical ☐ Administrative ☐ Payment Medical Policy Statements prepared by CSMG Co. and its affiliates (including CareSource) are derived from literature based on and supported by clinical guidelines, nationally recognized utilization and technology assessment guidelines, other medical management industry standards, and published MCO clinical policy guidelines. Medically necessary services include, but are not limited to, those health care services or supplies that are proper and necessary for the diagnosis or treatment of disease, illness, or injury and without which the patient can be expected to suffer prolonged, increased or new morbidity, impairment of function, dysfunction of a body organ or part, or significant pain and discomfort. These services meet the standards of good medical practice in the local area, are the lowest cost alternative, and are not provided mainly for the convenience of the member or provider. Medically necessary services also include those services defined in any Evidence of Coverage documents, Medical Policy Statements, Provider Manuals, Member Handbooks, and/or other policies and procedures. Medical Policy Statements prepared by CSMG Co. and its affiliates (including CareSource) do not ensure an authorization or payment of services. Please refer to the plan contract (often referred to as the Evidence of Coverage) for the service(s) referenced in the Medical Policy Statement. If there is a conflict between the Medical Policy Statement and the plan contract (i.e., Evidence of Coverage), then the plan contract (i.e., Evidence of Coverage) will be the controlling document used to make the determination.
  • Asfotase Alfa for Infants and Young Children with Hypophosphatasia: 7 Year Outcomes of a Single-Arm, Open-Label, Phase 2 Extension Trial

    Asfotase Alfa for Infants and Young Children with Hypophosphatasia: 7 Year Outcomes of a Single-Arm, Open-Label, Phase 2 Extension Trial

    Articles Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial Michael P Whyte, Jill H Simmons, Scott Moseley, Kenji P Fujita, Nicholas Bishop, Nada J Salman, John Taylor, Dawn Phillips, Mairead McGinn, William H McAlister Summary Background Our previous phase 2, open-label study of 11 infants and young children with life-threatening perinatal or Lancet Diabetes Endocrinol infantile hypophosphatasia showed 1 year safety and efficacy of asfotase alfa, an enzyme replacement therapy. We 2019; 7: 93–105 aimed to report the long-term outcomes over approximately 7 years of treatment. Published Online December 14, 2018 http://dx.doi.org/10.1016/ Methods We did a prespecified, end of study, 7 year follow-up of our single-arm, open-label, phase 2 trial in which S2213-8587(18)30307-3 children aged 3 years or younger with life-threatening perinatal or infantile hypophosphatasia were recruited from This online publication has been ten hospitals (six in the USA, two in the UK, one in Canada, and one in the United Arab Emirates). Patients received corrected. The corrected version asfotase alfa (1 mg/kg three times per week subcutaneously, adjusted to 3 mg/kg three times per week if required) for first appeared at thelancet. up to 7 years (primary treatment period plus extension phase) or until the product became commercially available; com/diabetes-endocrinology on January 22, 2019 dosage adjustments were made at each visit according to changes in the patient’s weight. The primary objectives of See Comment page 76 this extension study were to assess the long-term tolerability of asfotase alfa, defined as the number of patients with Center for Metabolic Bone one or more treatment-emergent adverse events, and skeletal manifestations associated with hypophosphatasia, Disease and Molecular evaluated using the Radiographic Global Impression of Change (RGI-C) scale (−3 indicating severe worsening, and Research, Shriners Hospital for +3 complete or near-complete healing).
  • BCBSVT Specialty Drug List Effective 2021.07.01.Xlsx

    BCBSVT Specialty Drug List Effective 2021.07.01.Xlsx

    Effective Date: 07/01/2021 SPECIALTY DRUG LIST Revised Date: 05/07/2021 DOSAGE EXCLUDED ON NATIONAL DRUG CLASS DRUG NAME GENERIC NAME FORM PERFORMANCE FORMULARY ANEMIA ARANESP SOLN DARBEPOETIN ALFA SOLN INJ ANEMIA ARANESP SOSY DARBEPOETIN ALFA SOLN PREFILLED SYRINGE ANEMIA EPOGEN SOLN EPOETIN ALFA INJ X ANEMIA PROCRIT SOLN EPOETIN ALFA INJ X ANEMIA REBLOZYL SOLR LUSPATERCEPT-AAMT FOR SUBCUTANEOUS INJ ANEMIA RETACRIT SOLN EPOETIN ALFA-EPBX INJ ANTI-GOUT AGENT KRYSTEXXA SOLN PEGLOTICASE INJ (FOR IV INFUSION) ANTI-INFECTIVE PREVYMIS SOLN LETERMOVIR IV SOLN ANTI-INFECTIVE PREVYMIS TABS LETERMOVIR TAB ASTHMA CINQAIR SOLN RESLIZUMAB IV INFUSION SOLN ASTHMA FASENRA SOSY BENRALIZUMAB SUBCUTANEOUS SOLN PREFILLED SYRINGE ASTHMA FASENRA PEN SOAJ BENRALIZUMAB SUBCUTANEOUS SOLN AUTO-INJECTOR ASTHMA NUCALA SOAJ MEPOLIZUMAB SUBCUTANEOUS SOLUTION AUTO-INJECTOR ASTHMA NUCALA SOLR MEPOLIZUMAB FOR INJ ASTHMA NUCALA SOSY MEPOLIZUMAB SUBCUTANEOUS SOLUTION PREF SYRINGE ASTHMA XOLAIR SOLR OMALIZUMAB FOR INJ ASTHMA XOLAIR SOSY OMALIZUMAB SUBCUTANEOUS SOLN PREFILLED SYRINGE CARDIOVASCULAR VYNDAMAX CAPS TAFAMIDIS CAP CARDIOVASCULAR VYNDAQEL CAPS TAFAMIDIS MEGLUMINE (CARDIAC) CAP CENTRAL NERVOUS SYSTEM AGENTS AUSTEDO TABS DEUTETRABENAZINE TAB CENTRAL NERVOUS SYSTEM AGENTS ENSPRYNG SOSY SATRALIZUMAB-MWGE SUBCUTANEOUS SOLN PREF SYRINGE CENTRAL NERVOUS SYSTEM AGENTS HETLIOZ CAPS TASIMELTEON CAPSULE CENTRAL NERVOUS SYSTEM AGENTS HETLIOZ LQ SUSP TASIMELTEON ORAL SUSP CHEMOTHERAPY PROTECTANT AMIFOSTINE SOLR AMIFOSTINE CRYSTALLINE FOR INJ CHEMOTHERAPY PROTECTANT ELITEK
  • Cipaglucosidase Alfa/Miglustat Phase 3 PROPEL Results

    Cipaglucosidase Alfa/Miglustat Phase 3 PROPEL Results

    AT-GAA (ATB200/AT2221) Phase 3 PROPEL Topline Results February 11, 2021 Phase 3 PROPEL Topline Results 2 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to top-line data from a global Phase 3 study to investigate AT-GAA for the treatment of Pompe Disease and the potential implications on these data for the future advancement and development of AT-GAA. Words such as, but not limited to, “look forward t o ,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “c o u l d ,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully manufacture and commercialize AT-GAA. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and Quarterly Report on 10-Q for the Quarter ended September 30, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.
  • Asfotase Alfa (Strensiq) for Treatment of Hypophosphatasia in Infants and Children

    Asfotase Alfa (Strensiq) for Treatment of Hypophosphatasia in Infants and Children

    AHRQ Healthcare Horizon Scanning System – Potential High-Impact Interventions Report Priority Area 08: Functional Limitations and Disability Prepared for: Agency for Healthcare Research and Quality U.S. Department of Health and Human Services 5600 Fishers Lane Rockville, MD 20857 www.ahrq.gov Contract No. HHSA290-2010-00006-C Prepared by: ECRI Institute 5200 Butler Pike Plymouth Meeting, PA 19462 December 2015 Statement of Funding and Purpose This report incorporates data collected during implementation of the Agency for Healthcare Research and Quality (AHRQ) Healthcare Horizon Scanning System by ECRI Institute under contract to AHRQ, Rockville, MD (Contract No. HHSA290-2010-00006-C). The findings and conclusions in this document are those of the authors, who are responsible for its content, and do not necessarily represent the views of AHRQ. No statement in this report should be construed as an official position of AHRQ or of the U.S. Department of Health and Human Services. This report’s content should not be construed as either endorsements or rejections of specific interventions. As topics are entered into the System, individual topic profiles are developed for technologies and programs that appear to be close to diffusion into practice in the United States. Those reports are sent to various experts with clinical, health systems, health administration, and/or research backgrounds for comment and opinions about potential for impact. The comments and opinions received are then considered and synthesized by ECRI Institute to identify interventions that experts deemed, through the comment process, to have potential for high impact. Please see the methods section for more details about this process.
  • Specialty Drug Benefit Document

    Specialty Drug Benefit Document

    Louisiana Healthcare Connections Specialty Drug Benefit ouisiana Healthcare Connections provides coverage of a number of specialty drugs. All specialty drugs, such as biopharmaceuticals and injectables, require a prior authorization (PA) to be approved for L payment by Louisiana Healthcare Connections. PA requirements are programmed specific to the drug. Since the list of specialty drugs changes over time due to new drug arrivals and other market conditions, it is important to contact Provider Services at 1-866-595-8133 or check the Louisiana Healthcare Connections website at www.LouisianaHealthConnect.com for updates to this benefit. Requests for specialty drugs can be submitted to Louisiana Healthcare Connections by filling out the Medication Prior Authorization Form that is available on the Louisiana Healthcare Connections website at www.LouisianaHealthConnect.com and faxing the request as instructed on the form. Louisiana Healthcare Connections members can receive the specialty drugs they require at any outpatient pharmacy enrolled in our pharmacy network that can supply specialty drugs. Providers that wish to have drugs distributed by a SPECIALTY PHARMACY should FAX the request to 1-866-399-0929 for review. If a provider wishes to dispense a specialty drug from OFFICE STOCK, the provider should FAX the request to Louisiana Healthcare Connections at 1-877-401-8172 for review. BRAND NAME INGREDIENTS SPECIAL INSTRUCTIONS ACTEMRA TOCILIZUMAB ACTHAR HP CORTICOTROPIN ACTIMMUNE INTERFERON GAMMA-1B ADAGEN PEGADEMASE BOVINE Limited Distribution
  • Myozyme, INN-Alglucosidase Alfa

    Myozyme, INN-Alglucosidase Alfa

    ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 1 1. NAME OF THE MEDICINAL PRODUCT Myozyme 50 mg powder for concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION One vial contains 50 mg of alglucosidase alfa. After reconstitution, the solution contains 5 mg of alglucosidase alfa* per ml and after dilution, the concentration varies from 0.5 mg to 4 mg/ml. *Human acid α-glucosidase is produced in Chinese hamster ovary cells (CHO) by recombinant DNA technology. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Powder for concentrate for solution for infusion. White to off-white powder. 4. CLINICAL PARTICULARS 4.1 Therapeutic indications Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid -glucosidase deficiency). Myozyme is indicated in adults and paediatric patients of all ages. 4.2 Posology and method of administration Myozyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases. Posology The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body weight administered once every 2 weeks. Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease. Paediatric and older people There is no evidence for special considerations when Myozyme is administered to paediatric patients of all ages or older people. Patients with renal and hepatic impairment The safety and efficacy of Myozyme in patients with renal or hepatic impairment have not been evaluated and no specific dose regimen can be recommended for these patients.