DOCSLIB.ORG

CADTH ISSUES in EMERGING HEALTH TECHNOLOGIES Informing Decisions About New Health Technologies

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
CADTH ISSUES in EMERGING HEALTH TECHNOLOGIES Informing Decisions About New Health Technologies CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES Informing Decisions About New Health Technologies Issue Mar 171 2018 Gene Therapy: An Overview of Approved and Pipeline Technologies CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES 1 Authors: Alison Sinclair, Saadul Islam, Sarah Jones Cite as: Gene therapy: an overview of approved and pipeline technologies. Ottawa: CADTH; 2018 Mar. (CADTH issues in emerging health technologies; issue 171). Acknowledgments: Louis de Léséleuc, Jeff Mason, Teo Quay, Joanne Kim, Lesley Dunfield, Eftyhia Helis, Iryna Magega, Jane Hurge ISSN: 1488-6324 (online) Disclaimer: The information in this document is intended to help Canadian health care decision-makers, health care professionals, health systems leaders, and policy- makers make well-informed decisions and thereby improve the quality of health care services. While patients and others may access this document, the document is made available for informational purposes only and no representations or warranties are made with respect to its fitness for any particular purpose. The information in this document should not be used as a substitute for professional medical advice or as a substitute for the application of clinical judgment in respect of the care of a particular patient or other professional judgment in any decision-making process. The Canadian Agency for Drugs and Technologies in Health (CADTH) does not endorse any information, drugs, therapies, treatments, products, processes, or services. While CADTH has taken care to ensure that the information prepared by it in this document is accurate, complete, and up-to-date as at the applicable date the material was first published by CADTH, CADTH does not make any guarantees to that effect. CADTH does not guarantee and is not responsible for the quality, currency, propriety, accuracy, or reasonableness of any statements, information, or conclusions contained in any third-party materials used in preparing this document. The views and opinions of third parties published in this document do not necessarily state or reflect those of CADTH. CADTH is not responsible for any errors, omissions, injury, loss, or damage arising from or relating to the use (or misuse) of any information, statements, or conclusions contained in or implied by the contents of this document or any of the source materials. This document may contain links to third-party websites. CADTH does not have control over the content of such sites. Use of third-party sites is governed by the third-party website owners’ own terms and conditions set out for such sites. CADTH does not make any guarantee with respect to any information contained on such third-party sites and CADTH is not responsible for any injury, loss, or damage suffered as a result of using such third-party sites. CADTH has no responsibility for the collection, use, and disclosure of personal information by third-party sites. Subject to the aforementioned limitations, the views expressed herein are those of CADTH and do not necessarily represent the views of Canada’s federal, provincial, or territorial governments. This document is prepared and intended for use in the context of the Canadian health care system. The use of this document outside of Canada is done so at the user’s own risk. This disclaimer and any questions or matters of any nature arising from or relating to the content or use (or misuse) of this document will be governed by and interpreted in accordance with the laws of the Province of Ontario and the laws of Canada applicable therein, and all proceedings shall be subject to the exclusive jurisdiction of the courts of the Province of Ontario, Canada. The copyright and other intellectual property rights in this document are owned by CADTH and its licensors. These rights are protected by the Canadian Copyright Act and other national and international laws and agreements. You are permitted to make copies of this document for non-commercial purposes only, provided it is not modified when reproduced and appropriate credit is given to CADTH and its licensors. About CADTH: CADTH is an independent, not-for-profit organization responsible for providing Canada’s health care decision-makers with objective evidence to help make informed decisions about the optimal use of drugs, medical devices, diagnostics, and procedures in our health care system. Funding: CADTH receives funding from Canada’s federal, provincial, and territorial governments, with the exception of Quebec. Contact [email protected] with inquiries about this notice or legal matters relating to CADTH services. CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES 2 Summary • Gene therapy is an area of therapeutics aimed at curing, or significantly improving the management of, diseases with few or no treatment alternatives. • A large proportion of the candidates for gene therapy include advanced-stage cancer or hematological conditions. In addition, rare or inherited disorders are also frequent targets of gene therapy. • While gene therapy developments are still largely in the research stage, companies are increasingly investing in these technologies. Recently, a number of products have been approved outside of Canada or are in the advanced stage of clinical research. • The upfront current cost of gene therapy is generally very high. Multi-stakeholder dialogues around management of cost and reimbursement of these products are necessary. • Specialized manufacturing facilities, care centres, and clinicians trained to conduct customized procedures for such therapies are vital to ensure accessibility and quality of care. Scope Methods This bulletin summarizes information on gene therapies that These bulletins are not systematic reviews of the literature and have been marketed in at least one country or are currently at do not involve a detailed critical appraisal of identified studies. an advanced or priority stage of development and are therefore They are not intended to provide recommendations for or most likely to appear on the market in the next two to three against a particular technology. years. General aspects of adoption and implementation are considered, but clinical and cost-effectiveness of individual Literature Search therapies are not reviewed. Details on mechanisms, processes A limited literature search was conducted on key resources, related to manufacturing and administration, and regulatory including PubMed, the Cochrane Library, University of York aspects are not within the scope of this bulletin. Centre for Reviews and Dissemination databases, and Canadian and major international health technology agencies. As well, a A comprehensive list of ongoing clinical trials on gene therapy focused Internet search was conducted. Methodological filters around the world or in Canada is also not within the scope of were applied to limit retrieval to randomized controlled trials, this bulletin. This information may be found at ClinicalTrials.gov, controlled clinical trials, and clinical studies. Where possible, 1 maintained by the US National Library of Medicine, and Health retrieval was limited to the human population. The search was 2 Canada’s Clinical Trials Database. also limited to English-language documents published between CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES 3 January 1, 2016, and December 8, 2017. Regular alerts updated the search until project completion; only citations retrieved Background before January 30, 2018, were incorporated into the report. What is Gene Therapy? According to the FDA, gene therapy is “the administration of Study Selection genetic material to modify or manipulate the expression of The search results were divided between two authors by topic a gene product or to alter the biological properties of living area. No duplicate screening was completed. In addition to cells for therapeutic use.”3 In Canada, gene therapies are the studies identified by the literature search, publications of included in the definition of “drug” under theFood and Drugs interest cited by identified sources were also included in the Act and are regulated under the Food and Drug Regulations.4-6 report. As the report was intended to focus on the therapies Both the US and Canada regulate gene therapies as biologic most likely to appear on the market in the next two to three drug therapies.3-5,7 Other jurisdictions, such as Europe, have a years, journal articles, database entries, or Web pages were separate regulatory pathway for gene therapies.7,8 considered for inclusion if they provided information on a Gene therapy involves the administration of specific genetic gene therapy that has been approved for marketing anywhere material (i.e., DNA or RNA) via a carrier, known as a “vector,” that in the world or was in active development, either in phase III enables the foreign genetic material to enter the target cells.9 or in earlier phases with a special regulatory designation. All Most gene therapies use modified versions of natural viruses indications were eligible. as vectors, as they are an efficient way of introducing DNA or RNA into a cell.8-10 The gene therapy agent can be injected Stakeholder Review into the body (in vivo gene therapy) or used to modify cells A draft version of this bulletin was posted publicly for taken from the body, which are then re-infused (ex vivo gene stakeholder review. therapy; Figure 1). Replacement gene therapy aims to provide a working copy of the damaged gene(s), boost the availability Figure 1: Schematic of Approaches to Gene Therapy Applicable to Both Gene Editing and
Load more

Recommended publications
  • Luxturna Voretigene Neparvovec Rzyl Molina Clinical Policy
    Subject: Luxturna (Voretigene neparvovec-rzyl) Original Effective Date: 7/10/2018 Policy Number: MCP-318 Revision Date(s): Review Date(s): 7/10/2018 DISCLAIMER This Molina Clinical Policy (MCP) is intended to facilitate the Utilization Management process. It expresses Molina's determination as to whether certain services or supplies are medically necessary, experimental, investigational, or cosmetic for purposes of determining appropriateness of payment. The conclusion that a particular service or supply is medically necessary does not constitute a representation or warranty that this service or supply is covered (i.e., will be paid for by Molina) for a particular member. The member's benefit plan determines coverage. Each benefit plan defines which services are covered, which are excluded, and which are subject to dollar caps or other limits. Members and their providers will need to consult the member's benefit plan to determine if there are any exclusion(s) or other benefit limitations applicable to this service or supply. If there is a discrepancy between this policy and a member's plan of benefits, the benefits plan will govern. In addition, coverage may be mandated by applicable legal requirements of a State, the Federal government or CMS for Medicare and Medicaid members. CMS's Coverage Database can be found on the CMS website. The coverage directive(s) and criteria from an existing National Coverage Determination (NCD) or Local Coverage Determination (LCD) will supersede the contents of this MCP document and provide the directive for all Medicare members. SUMMARY OF EVIDENCE/POSITION This policy addresses the coverage of Luxturna (Voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of individuals with confirmed bi-allelic RPE65 mutation-associated retinal dystrophy, when appropriate criteria are met.
    [Show full text]
  • Luxturna, INN-Voretigene Neparvovec
    20 September 2018 EMA/CHMP/700911/2018 Committee for Medicinal Products for Human Use (CHMP) Assessment report Luxturna International non-proprietary name: voretigene neparvovec Procedure No. EMEA/H/C/004451/0000 Note Assessment report as adopted by the CHMP with all information of a commercially confidential nature deleted. 30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact An agency of the European Union © European Medicines Agency, 2019. Reproduction is authorised provided the source is acknowledged. Table of contents 1. Background information on the procedure .............................................. 6 1.1. Submission of the dossier ...................................................................................... 6 1.2. Steps taken for the assessment of the product ......................................................... 7 2. Scientific discussion ................................................................................ 9 2.1. Problem statement ............................................................................................... 9 2.1.1. Disease or condition ........................................................................................... 9 2.1.2. Epidemiology .................................................................................................... 9 2.1.3. Biologic features ...............................................................................................
    [Show full text]
  • Spark Therapeutics and Pfizer Announce That SPK-9001, An
    Spark Therapeutics and Pfizer Announce that SPK-9001, an Investigational Hemophilia B Medicine, has been Granted Access to the PRIority MEdicines (PRIME) Program by the European Medicines Agency PHILADELPHIA and NEW YORK, March 1, 2017 (GLOBE NEWSWIRE)— Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE) announced today that SPK-9001, the lead investigational candidate in the companies' SPK-FIX program has been granted support through the European Medicines Agency (EMA) PRIority MEdicines (PRIME) program. According to the EMA, PRIME is intended to enhance support for new treatments in development “that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.” “We are pleased that investigational SPK-9001 has been granted access to the PRIME program, which potentially will help accelerate the delivery of this potential gene therapy to European patients living with hemophilia B,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “We appreciate the EMA’s dedication to moving treatments forward for patients who are underserved with current options, and we look forward to our continued collaboration on SPK-9001.” SPK-9001 is an experimental treatment for hemophilia B that is currently being investigated in an ongoing Phase 1/2 trial as a potential single-dose therapy. The PRIME program provides enhanced interaction and early dialogue with developers of promising medicines, which is intended to optimize development plans and speed up evaluation so these medicines can reach patients earlier. SPK-9001 received orphan product designation from the U.S. Food and Drug Administration (FDA) in September 2015, and later received breakthrough therapy designation from the FDA in July 2016.
    [Show full text]
  • Clinical Guideline Luxturna (Voretigene Neparvovec-Rzyl)
    Clinical Guideline Guideline Number: CG060, Ver. 1 Luxturna (voretigene neparvovec-rzyl) Disclaimer Clinical guidelines are developed and adopted to establish evidence-based clinical criteria for utilization management decisions. Oscar may delegate utilization management decisions of certain services to third-party delegates, who may develop and adopt their own clinical criteria. Clinical guidelines are applicable to certain plans. Clinical guidelines are applicable to members enrolled in Medicare Advantage plans only if there are no criteria established for the specified service in a Centers for Medicare & Medicaid Services (CMS) national coverage determination (NCD) or local coverage determination (LCD) on the date of a prior authorization request. Services are subject to the terms, conditions, limitations of a member’s policy and applicable state and federal law. Please reference the member’s policy documents (e.g., Certificate/Evidence of Coverage, Schedule of Benefits) or contact Oscar at 855-672-2755 to confirm coverage and benefit conditions. Summary Luxturna was the first in vivo (within living cells) gene therapy approved by the FDA in December 2017 for children and adults with rare inherited vision disorders caused by mutated RPE65 gene. For individuals who have mutations in both copies of the RPE65 gene, they cannot make proteins in the eye that convert light, which leads to partial or total vision loss. Luxturna is delivered as a single-dose, subretinal injection in each eye that works by targeting these mutations to create proteins again for light detection. Subretinal injection occurs after complete vitrectomy; therefore members first must receive a pars plana vitrectomy. Definitions “Gene therapy” is a technique that replaces a mutated gene with a healthy gene, inactivates a mutated gene, or introduces a new gene that helps fight against diseases and disorders.
    [Show full text]
  • Gene Therapy for Inherited Retinal Diseases
    1278 Review Article on Novel Tools and Therapies for Ocular Regeneration Page 1 of 13 Gene therapy for inherited retinal diseases Yan Nuzbrokh1,2,3, Sara D. Ragi1,2, Stephen H. Tsang1,2,4 1Department of Ophthalmology, Edward S. Harkness Eye Institute, Columbia University Irving Medical Center, New York, NY, USA; 2Jonas Children’s Vision Care, New York, NY, USA; 3Renaissance School of Medicine at Stony Brook University, Stony Brook, New York, NY, USA; 4Department of Pathology & Cell Biology, Columbia University Irving Medical Center, New York, NY, USA Contributions: (I) Conception and design: All authors; (II) Administrative support: SH Tsang; (III) Provision of study materials or patients: SH Tsang; (IV) Collection and assembly of data: All authors; (V) Manuscript writing: All authors; (VI) Final approval of manuscript: All authors. Correspondence to: Stephen H. Tsang, MD, PhD. Harkness Eye Institute, Columbia University Medical Center, 635 West 165th Street, Box 212, New York, NY 10032, USA. Email: [email protected]. Abstract: Inherited retinal diseases (IRDs) are a genetically variable collection of devastating disorders that lead to significant visual impairment. Advances in genetic characterization over the past two decades have allowed identification of over 260 causative mutations associated with inherited retinal disorders. Thought to be incurable, gene supplementation therapy offers great promise in treating various forms of these blinding conditions. In gene replacement therapy, a disease-causing gene is replaced with a functional copy of the gene. These therapies are designed to slow disease progression and hopefully restore visual function. Gene therapies are typically delivered to target retinal cells by subretinal (SR) or intravitreal (IVT) injection.
    [Show full text]
  • First Gene Therapy Fda-Approved for An
    s FEATURE FIRST GENE THERAPY FDA-APPROVED FOR AN INHERITED RETINAL DISEASE The approval has stimulated research into gene therapies for other IRDs. BY MEGHAN J. DEBENEDICTIS, MS, LGC, MED, AND ALEKSANDRA V. RACHITSKAYA, MD he idea of gene therapy has been for clustered regularly interspaced short Scientists and companies have discussed in the medical litera- palindromic repeats) could allow edit- spent decades perfecting the use ture since as early as the 1970s. In ing of one or several sites within the of vectors for genetic material and 1972, Friedman and Roblin pro- mammalian genome.5 identifying ways to deliver them to posed that it was theoretically The most common type of gene increase therapeutic efficacy and possibleT to introduce “good” DNA to therapy is replacement gene therapy, treatment duration. Early investiga- replace defective DNA.1 Over the years, which involves replacing a mutated tions used viruses that delivered a number of gene therapy clinical trials gene that causes disease with a genes to every cell in the body, which emerged in efforts to treat genetic dis- healthy copy of that gene. It is first triggered a massive immune response eases of inborn errors of metabolism, necessary to identify the causative that could lead to organ failure. More all with varying degrees of success. mutated gene. This technology recently designed vectors deliver The basic principle of gene therapy works best in autosomal recessive specific genes to specific cells. is to put corrective genetic material biallelic disease with loss-of-function into cells to treat genetic disease. mutations. A vector is then created TARGET: IRDS Several gene therapy approaches, to carry a wild-type copy of the With the eye’s unique immunologic including replacement gene therapy, gene into the cell of interest.
    [Show full text]
  • Gene Therapy for Inherited Retinal Dystrophy, 2.04.144
    MEDICAL POLICY – 2.04.144 Gene Therapy for Inherited Retinal Dystrophy BCBSA Ref. Policy: 2.04.144 Effective Date: Mar. 1, 2021 RELATED MEDICAL POLICIES: Last Revised: Feb. 18, 2021 None Replaces: 8.01.536 Select a hyperlink below to be directed to that section. POLICY CRITERIA | DOCUMENTATION REQUIREMENTS | CODING RELATED INFORMATION | EVIDENCE REVIEW | REFERENCES | HISTORY ∞ Clicking this icon returns you to the hyperlinks menu above. Introduction The retina is found at the back of the eye. It is made up of several layers. One of these layers contains cells called rods and cones. The rods and cones are stimulated when light enters our eyes. They convert the light energy into chemicals, which then create an electrical signal. The optic nerve sends the electrical signal to the brain. Many steps are required for this process to work correctly. One of these steps involves a protein called RPE65. This protein helps make some of the chemical changes that happen in the retina. A specific gene tells the body how to make this protein. If that gene is not normal, the protein cannot be made. The person without this protein will have visual problems called retinal dystrophy and can become blind, even at an early age. Changes in the RPE65 gene are rare. A new treatment uses an engineered virus to insert a healthy copy of the gene into the retinal cells. This treatment requires a genetic test to confirm the specific type of retinal dystrophy. Treatment also requires a specific amount of healthy retina to be available. This policy describes when this treatment may be considered medically necessary.
    [Show full text]
  • Twenty-Five Years of Gene Therapy for ADA-SCID: from Bubble Babies to an Approved Drug
    Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug Francesca Ferrua1,2 and Alessandro Aiuti1,2,* 1San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;2Vita-Salute San Raffaele University, Milan, Italy. Twenty-five years have passed since first attempts of gene therapy (GT) in children affected by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect, also known by the general public as bubble babies. ADA-SCID is fatal early in life if untreated. Unconditioned hematopoietic stem cell (HSC) transplant from matched sibling donor represents a curative treatment but is available for few patients. Enzyme replacement therapy can be life-saving, but its chronic use has many drawbacks. This review summarizes the history of ADA-SCID GT over the last 25 years, starting from first pioneering studies in the early 1990s using gamma-retroviral vectors, based on multiple infusions of genetically corrected autologous peripheral blood lymphocytes. HSC represented the ideal target for gene correction to guarantee production of engineered multi-lineage progeny, but it required a decade to achieve thera- peutic benefit with this approach. Introduction of low-intensity conditioning represented a crucial step in achieving stable gene-corrected HSC engraftment and therapeutic levels of ADA-expressing cells. Recent clinical trials demonstrated that gamma-retroviral GT for ADA-SCID has a favorable safety profile and is effective in restoring normal purine metabolism and immune functions in patients >13 years after treatment. No abnormal clonal proliferation or leukemia development have been observed in >40 patients treated experimentally in five different centers worldwide.
    [Show full text]
  • Voretigene Neparvovec-Rzyl (Luxturna) Policy Number: 249 Effective Date: 06/01/2021 Last Review: 04/22/2021 Next Review Date: 04/22/2022
    MEDICAL COVERAGE POLICY SERVICE: Voretigene Neparvovec-rzyl (Luxturna) Policy Number: 249 Effective Date: 06/01/2021 Last Review: 04/22/2021 Next Review Date: 04/22/2022 Important note Even though this policy may indicate that a particular service or supply may be considered covered, this conclusion is not based upon the terms of your particular benefit plan. Each benefit plan contains its own specific provisions for coverage and exclusions. Not all benefits that are determined to be medically necessary will be covered benefits under the terms of your benefit plan. You need to consult the Evidence of Coverage to determine if there are any exclusions or other benefit limitations applicable to this service or supply. If there is a discrepancy between this policy and your plan of benefits, the provisions of your benefits plan will govern. However, applicable state mandates will take precedence with respect to fully insured plans and self- funded non-ERISA (e.g., government, school boards, church) plans. Unless otherwise specifically excluded, Federal mandates will apply to all plans. With respect to Senior Care members, this policy will apply unless Medicare policies extend coverage beyond this Medical Policy & Criteria Statement. Senior Care policies will only apply to benefits paid for under Medicare rules, and not to any other health benefit plan benefits. CMS's Coverage Issues Manual can be found on the CMS website. SERVICE: Voretigene Neparvovec-rzyl (Luxturna™) PRIOR AUTHORIZATION: Required. POLICY: For Medicaid plans, please confirm coverage as outlined in the Texas Medicaid TMPPM. For all other plans, SWHP may find Voretigene Neparvovec-rzyl medically necessary in the treatment of inherited retinal diseases when the following conditions are met: Member is between 12 months and 65 years of age; AND Has diagnosis of a confirmed biallelic RPE65 mutation-associated retinal dystrophy (e.g.
    [Show full text]
  • Landscape Review and Evidence Map of Gene Therapy, Part 2
    EMERGING TECHNOLOGIES AND THERAPEUTICS REPORT 1 Landscape Review and Evidence Map of Gene Therapy, Part II: Chimeric Antigen Receptor- T cell (CAR-T), Autologous Cell, Antisense, RNA Interference (RNAi), Zinc Finger Nuclease (ZFN), Genetically Modified Oncolytic Herpes Virus Andrea Richardson, Eric Apaydin, Sangita Baxi, Jerry Vockley, Olamigoke Akinniranye, Rachel Ross, Jody Larkin, Aneesa Motala, Gulrez Azhar, and Susanne Hempel RAND Corporation June 2019 Revision [February 2020] Since the completion of this report a gene therapy herein described as AVXS-101 has been approved by the FDA as Zolgensma therefore the report was revised in November 2019 to reflect that change. All statements, findings, and conclusions in this publication are solely those of the authors and do not necessarily represent the views of the Patient-Centered Outcomes Research Institute (PCORI) or its Board of Governors. This publication was developed through a contract to support PCORI's work. Questions or comments may be sent to PCORI at [email protected] or by mail to Suite 900, 1828 L Street, NW, Washington, DC 20036. ©2019 Patient-Centered Outcomes Research Institute. For more information see https://www.pcori.org EMERGING TECHNOLOGIES AND THERAPEUTICS REPORT 2 Table of Contents Executive Summary ......................................................................................................... 6 Introduction of Report Series .......................................................................................... 8 History of Gene Therapies ...........................................................................................
    [Show full text]
  • Luxturna: FDA Documents Reveal the Value of a Costly Gene Therapy
    Drug Discovery Today Volume 24, Number 4 April 2019 PERSPECTIVE feature Luxturna: FDA documents reveal the value of a costly gene therapy Jonathan J. Darrow In 2017, the US Food and Drug Administration (FDA) approved voretigene neparvovec-rzyl (Luxturna), a gene therapy used to treat a rare form of inherited blindness. Widely described by the media as a curative treatment that ‘restores vision’, it was priced at US$850 000. Although voretigene neparvovec-rzyl represents a substantial therapeutic advance, most reports have failed to adequately describe study outcomes as documented by FDA reviewers. These documents reveal that the drug is not expected to restore normal vision, that only about half of treated patients met the FDA’s threshold for minimally meaningful improvement, that improvements might not persist long-term, that the most common PERSPECTIVE measure of visual function was rejected as a primary endpoint after yielding mixed results, and that two patients experienced permanent vision loss. Over US$100 million of additional publicly-funded costs are not evident from the US$850 000 figure. Features Introduction that targets a disease caused by mutations in a while looking through a tunnel. Other patients Voretigene neparvovec-rzyl (Luxturna) was ap- specific gene [1]. or their family members reported improvements proved by the FDA in December 2017 for the The transformative nature of voretigene such as the ability to read large print rather than treatment of biallelic retinal pigment epitheli- neparvovec-rzyl has been widely lauded by Braille, eat at dimly lit restaurants, use iPhone um-specific 65 kilodalton (RPE65) mutation-as- regulators, patients, and the media.
    [Show full text]
  • Luxturna™ (Voretigene Neparvovec-Rzyl)
    UnitedHealthcare® Commercial Medical Benefit Drug Policy Luxturna™ (Voretigene Neparvovec-Rzyl) Policy Number: 2020D0063F Effective Date: December 1, 2020 Instructions for Use Table of Contents Page Community Plan Policy Coverage Rationale ....................................................................... 1 • Luxturna™ (Voretigene Neparvovec-Rzyl) Documentation Requirements ...................................................... 1 Applicable Codes .......................................................................... 2 Background.................................................................................... 2 Benefit Considerations .................................................................. 2 Clinical Evidence ........................................................................... 3 U.S. Food and Drug Administration ............................................. 3 Centers for Medicare and Medicaid Services ............................. 3 References ..................................................................................... 3 Policy History/Revision Information ............................................. 4 Instructions for Use ....................................................................... 4 Coverage Rationale See Benefit Considerations Luxturna is proven and/or medically necessary for the treatment of Inherited Retinal Dystrophies (IRD) caused by mutations in the retinal pigment epithelium-specific protein 65kDa (RPE65) gene in patients who meet all of the following criteria:1-2 Patient is
    [Show full text]