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Chapter 7: Monogenic Forms of Diabetes
CHAPTER 7 MONOGENIC FORMS OF DIABETES Mark A. Sperling, MD, and Abhimanyu Garg, MD Dr. Mark A. Sperling is Emeritus Professor and Chair, University of Pittsburgh, Department of Pediatrics, Children’s Hospital of Pittsburgh of UPMC, Pittsburgh, PA. Dr. Abhimanyu Garg is Professor of Internal Medicine and Chief of the Division of Nutrition and Metabolic Diseases at University of Texas Southwestern Medical Center, Dallas, TX. SUMMARY Types 1 and 2 diabetes have multiple and complex genetic influences that interact with environmental triggers, such as viral infections or nutritional excesses, to result in their respective phenotypes: young, lean, and insulin-dependence for type 1 diabetes patients or older, overweight, and often manageable by lifestyle interventions and oral medications for type 2 diabetes patients. A small subset of patients, comprising ~2%–3% of all those diagnosed with diabetes, may have characteristics of either type 1 or type 2 diabetes but have single gene defects that interfere with insulin production, secretion, or action, resulting in clinical diabetes. These types of diabetes are known as MODY, originally defined as maturity-onset diabetes of youth, and severe early-onset forms, such as neonatal diabetes mellitus (NDM). Defects in genes involved in adipocyte development, differentiation, and death pathways cause lipodystrophy syndromes, which are also associated with insulin resistance and diabetes. Although these syndromes are considered rare, more awareness of these disorders and increased availability of genetic testing in clinical and research laboratories, as well as growing use of next generation, whole genome, or exome sequencing for clinically challenging phenotypes, are resulting in increased recognition. A correct diagnosis of MODY, NDM, or lipodystrophy syndromes has profound implications for treatment, genetic counseling, and prognosis. -
(Human Papillomavirus 9-Valent Vaccine, Recombinant), for Use In
tools Approved Drugs FDA has approved a supplemental new drug propelled, joystick-controlled, easy-to-use application (sNDA) for Xtandi® colonoscope system. • The Food and Drug Administration (FDA) (enzalutamide) capsules in advanced has approved a supplemental biologics prostate cancer. • Varian Medical Systems (varian.com) has license application (sBLA) for the use of received 510(k) clearance from the FDA to Arzerra® (ofatumumab) (Genmab A/S, market the Nexus DR, a high resolution genmab.com) in combination with Drugs in the News imaging system for X-ray imaging using a fludarabine and cyclophosphamide for the digital X-ray detector. treatment of patients with relapsed chronic • Genentech (gene.com) has received a lymphocytic leukemia (CLL). second breakthrough therapy designation Approved Genetic Tests & from the FDA for Alecensa® (alectinib) Assays • Amgen (amgen.com) announced that the for the treatment of adult patients with FDA has approved the sBLA for Blincyto® advanced ALK-positive NSCLC who have • AstraZeneca (astrazeneca-us.com) (blinatumomab) to include new data not received prior treatment with an ALK announced that the FDA has approved a supporting the treatment of pediatric patients inhibitor. blood-based companion diagnostic for with Philadelphia chromosome-negative (Ph-) Tagrisso® (osimertinib). relapsed or refractory B-cell precursor acute • AbbVie (abbvie.com) submitted an sNDA lymphoblastic leukemia (ALL). to the FDA for Imbruvica® (ibrutinib) to treat patients with marginal zone lymphoma, • Merck Sharp & Dohme Corp.’s (merck.com) a form of non-Hodgkin’s lymphoma. FDA Approves Two-Dose Keytruda® (pembrolizumab) has received Vaccination Regime FDA approval in first-line non-small cell lung • Fate Therapeutics, Inc. -
Study Protocol
Official Title: A MULTI-CENTRE RANDOMISED CLINICAL TRIAL OF BIOMARKER-DRIVEN MAINTENANCE TREATMENT FOR FIRST- LINE METASTATIC COLORECTAL CANCER (MODUL) NCT Number: NCT02291289 Document Date: Protocol Version 9: 18-Feb-2020 PROTOCOL TITLE: A MULTI-CENTRE RANDOMISED CLINICAL TRIAL OF BIOMARKER-DRIVEN MAINTENANCE TREATMENT FOR FIRST-LINE METASTATIC COLORECTAL CANCER (MODUL) PROTOCOL NUMBER: MO29112 VERSION NUMBER: 9 EUDRACT NUMBER: 2014-001017-61 IND NUMBER: N/A TEST PRODUCT: Atezolizumab (MPDL3280A, RO5541267) Bevacizumab (RO4876646) Cobimetinib (RO5514041) Pertuzumab (RO4368451) Trastuzumab (RO0452317) Vemurafenib (RO5185426) Cetuximab And combinations thereof MEDICAL MONITOR: Dr. SPONSOR: F. Hoffmann-La Roche Ltd. DATE FINAL: See electronic date stamp below FINAL PROTOCOL APPROVAL Date and Time (UTC) Title Approver's Name 18-Feb-2020 15:44:34 Company Signatory CONFIDENTIAL This clinical study is being sponsored globally by F. Hoffmann-La Roche Ltd of Basel, Switzerland. However, it may be implemented in individual countries by Roche’s local affiliates, including Genentech, Inc. in the United States. The information contained in this document, especially any unpublished data, is the property of F. Hoffmann-La Roche Ltd (or under its control) and therefore is provided to you in confidence as an investigator, potential investigator, or consultant, for review by you, your staff, and an applicable Ethics Committee or Institutional Review Board. It is understood that this information will not be disclosed to others without written authorization from Roche except to the extent necessary to obtain informed consent from persons to whom the drug may be administered. Bevacizumab — F. Hoffmann-La Roche Ltd. Protocol MO29112, Version 9 DATES AMENDED: Version 1: 5 August 2014 Version 2: 29 October 2014 Version 3: 2 February 2015 Version 4: 30 November 2015 Version 5: 11 April 2016 Version 6: 24 November 2016 Version 7: 8 August 2018 Version 8: 19 December 2018 Bevacizumab — F. -
Myalept® (Metreleptin)
UnitedHealthcare Pharmacy Clinical Pharmacy Programs Program Number 2021 P 2032-11 Program Prior Authorization/Medical Necessity Medication Myalept® (metreleptin) P&T Approval Date 5/2014, 7/2014, 8/2014, 7/2015, 6/2016, 5/2017, 5/2018, 5/2019, 5/2020, 5/2021 Effective Date 8/1/2021; Oxford only: 8/1/2021 1. Background: Myalept (metreleptin) is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.1 Myalept is available only through a restricted distribution program under a Risk Evaluation and Mitigation Strategy (REMS), called the Myalept REMS program, because of the risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and the risk of lymphoma. 2. Coverage Criteria: A. Initial Authorization 1. Myalept will be approved based on all of the following criteria: a. Diagnosis of congenital or acquired generalized lipodystrophy associated with leptin deficiency -AND- b. Myalept is being used as an adjunct to diet modification -AND- c. Prescribed by an endocrinologist -AND- d. Documentation demonstrates that patient has at least one of the following: (1) Diabetes mellitus or insulin resistance with persistent hyperglycemia (HgbA1C > 7.0) despite both of the following: (a) Dietary intervention (b) Optimized insulin therapy at maximum tolerated doses © 2021 UnitedHealthcare Services, Inc. 1 -OR- (2) Persistent hypertriglyceridemia (TG > 250) despite both of the following: (a) Dietary intervention (b) Optimized therapy with at least two triglyceride-lowering agents from different classes (e.g., fibrates, statins) at maximum tolerated doses Authorization will be issued for 12 months B. -
Updated: May 20, 2021 Prior Authorization Drugs (NEW ADDITIONS HIGHLIGHTED)
Updated: May 20, 2021 Prior Authorization Drugs (NEW ADDITIONS HIGHLIGHTED) A Abatacept (ORENCIA) Abemaciclib (VERZENIO) Abiraterone Acetate (ZYTIGA) Abiraterone Acetate (+ GENERIC FORMULATIONS) AbobotulinumtoxinA (DYSPORT) Abraxane (ABRAXANE) Acalabrutinib (CALQUENCE) Actemra (ACTEMRA) Adalimumab (AMGEVITA) Adalimumab (HADLIMA) Adalimumab (HULIO) Adalimumab (HUMIRA) Adalimumab (HYRIMOZ) Adalimumab (IDACIO) Adcetris (ADCETRIS) Adcirca (ADCIRCA) Adempas (ADEMPAS) Afatinib (GIOTRIF) Afinitor (AFINITOR) Aflibercept (EYLEA) Aflibercept (ZALTRAP) Afstyla (AFSTYLA) Agalsidase Alfa (REPLAGAL) Agalsidase Beta (FABRAZYM) Aimovig (AIMOVIG) Ajovy (FREMANEZUMAB) Aldesleukin (PROLEUKIN) Aldurazyme (ALDURAZYME) Alecensaro (ALECENSARO) Alectinib (ALECENSARO) Alefacept (AMEVIVE) Alemtuzumab (MABCAMPATH) Alemtuzumab (LEMTRADA) Alglucosidase Alfa (MYOZYME) Alimta (ALIMTA) Alirocumab (PRALUENT) Alpha-1-Proteinase Inhibitor (PROLASTIN - C) Alpha-1-Proteinase Inhibitor (ZEMAIRA) Alpelisib (PIQRAY) Alunbrig (ALUNBRIG) Ambrisentan (VOLIBRIS) Ambrisentan (Generic Formulations) Amevive (AMEVIVE) Amgevita (ADALIMUMAB) Amifampridine Phosphate (FIRDAPSE) Amifampridine Phosphate (RUZURGI) Anakinra (KINERET) Apalutamide (ERLEADA) Apomorphine (KYNMOBI) Apremilast (OTEZLA) Page 1 of 17 Updated: May 20, 2021 Arsenic Trioxide (TRISENOX) Arsenic Trioxide (Generic Formulations) Arzerra (ARZERRA) Asfotase Alfa (STRENSIQ) Asparaginase (ERWINASE) Asunaprevir (SUNVEPRA) Atezolizumab (TECENTRIQ) Atriance (ATRIANCE) Aubagio (AUBAGIO) Avastin (AVASTIN) Avelumab (BAVENCIO) Avonex -
DRUGS REQUIRING PRIOR AUTHORIZATION in the MEDICAL BENEFIT Page 1
Effective Date: 08/01/2021 DRUGS REQUIRING PRIOR AUTHORIZATION IN THE MEDICAL BENEFIT Page 1 Therapeutic Category Drug Class Trade Name Generic Name HCPCS Procedure Code HCPCS Procedure Code Description Anti-infectives Antiretrovirals, HIV CABENUVA cabotegravir-rilpivirine C9077 Injection, cabotegravir and rilpivirine, 2mg/3mg Antithrombotic Agents von Willebrand Factor-Directed Antibody CABLIVI caplacizumab-yhdp C9047 Injection, caplacizumab-yhdp, 1 mg Cardiology Antilipemic EVKEEZA evinacumab-dgnb C9079 Injection, evinacumab-dgnb, 5 mg Cardiology Hemostatic Agent BERINERT c1 esterase J0597 Injection, C1 esterase inhibitor (human), Berinert, 10 units Cardiology Hemostatic Agent CINRYZE c1 esterase J0598 Injection, C1 esterase inhibitor (human), Cinryze, 10 units Cardiology Hemostatic Agent FIRAZYR icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent HAEGARDA c1 esterase J0599 Injection, C1 esterase inhibitor (human), (Haegarda), 10 units Cardiology Hemostatic Agent ICATIBANT (generic) icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent KALBITOR ecallantide J1290 Injection, ecallantide, 1 mg Cardiology Hemostatic Agent RUCONEST c1 esterase J0596 Injection, C1 esterase inhibitor (recombinant), Ruconest, 10 units Injection, lanadelumab-flyo, 1 mg (code may be used for Medicare when drug administered under Cardiology Hemostatic Agent TAKHZYRO lanadelumab-flyo J0593 direct supervision of a physician, not for use when drug is self-administered) Cardiology Pulmonary Arterial Hypertension EPOPROSTENOL (generic) -
761094Orig1s000
CENTER FOR DRUG EVALUATION AND RESEARCH APPLICATION NUMBER: 761094Orig1s000 CLINICAL REVIEW(S) Medical Officer’s Review of BLA 761094 Review #2 BLA 761094 Submission Date: July 19, 2018 Receipt Date: July 19, 2018 Review Date: July 23, 2018 Applicant: Dompé farmaceutici S.p.A. Via Santa Lucia 6-20122 Milan, Italy Applicant’s Representative: Lamberto Dionigi. Regulatory Affairs & Drug Safety Director 39-02-5838-3559 Drug: OXERVATE (cenegermin – bkbj) ophthalmic solution, 20 mcg/mL Submitted: Reference is made to the meeting Teleconference of 15-July-2018, in which the FDA has requested to provide information available at Dompé on the use of Oxervate in the pediatric population. Pediatric patients exposed to Oxervate: Dompé confirms that Oxervate is not yet approved in Europe for use in children and no clinical studies have been conducted in this population. As far as the company is aware, four (4) Neurotrophic Keratitis (NK) pediatric patients, aged from 2 to 12 years of age, have been exposed to Oxervate. A 6-year-old child with severe NK secondary to Stuve-Wiedmann syndrome was treated with Oxervate under “Temporary Authorization for Use” (ATU) in France. Dompé reports that the corneal ulcer was completely healed at the end of the treatment. There were signs of corneal sensitivity recovery by month 8 post-treatment. A 9-year-old child with NK was treated with Oxervate under ATU in France. Dompé reports that the lesion improved, but did not completely heal with the treatment. There is no other follow-up information available. A 12-year-old child with severe NK was treated with Oxervate under Expanded Access in the US. -
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 -
FLT3 Inhibitors in Acute Myeloid Leukemia Mei Wu1, Chuntuan Li2 and Xiongpeng Zhu2*
Wu et al. Journal of Hematology & Oncology (2018) 11:133 https://doi.org/10.1186/s13045-018-0675-4 REVIEW Open Access FLT3 inhibitors in acute myeloid leukemia Mei Wu1, Chuntuan Li2 and Xiongpeng Zhu2* Abstract FLT3 mutations are one of the most common findings in acute myeloid leukemia (AML). FLT3 inhibitors have been in active clinical development. Midostaurin as the first-in-class FLT3 inhibitor has been approved for treatment of patients with FLT3-mutated AML. In this review, we summarized the preclinical and clinical studies on new FLT3 inhibitors, including sorafenib, lestaurtinib, sunitinib, tandutinib, quizartinib, midostaurin, gilteritinib, crenolanib, cabozantinib, Sel24-B489, G-749, AMG 925, TTT-3002, and FF-10101. New generation FLT3 inhibitors and combination therapies may overcome resistance to first-generation agents. Keywords: FMS-like tyrosine kinase 3 inhibitors, Acute myeloid leukemia, Midostaurin, FLT3 Introduction RAS, MEK, and PI3K/AKT pathways [10], and ultim- Acute myeloid leukemia (AML) remains a highly resist- ately causes suppression of apoptosis and differentiation ant disease to conventional chemotherapy, with a me- of leukemic cells, including dysregulation of leukemic dian survival of only 4 months for relapsed and/or cell proliferation [11]. refractory disease [1]. Molecular profiling by PCR and Multiple FLT3 inhibitors are in clinical trials for treat- next-generation sequencing has revealed a variety of re- ing patients with FLT3/ITD-mutated AML. In this re- current gene mutations [2–4]. New agents are rapidly view, we summarized the preclinical and clinical studies emerging as targeted therapy for high-risk AML [5, 6]. on new FLT3 inhibitors, including sorafenib, lestaurtinib, In 1996, FMS-like tyrosine kinase 3/internal tandem du- sunitinib, tandutinib, quizartinib, midostaurin, gilteriti- plication (FLT3/ITD) was first recognized as a frequently nib, crenolanib, cabozantinib, Sel24-B489, G-749, AMG mutated gene in AML [7]. -
Keeping up with FDA Drug Approvals: 60 New Drugs in 60 Minutes Elizabeth A
Keeping Up with FDA Drug Approvals: 60 New Drugs in 60 Minutes Elizabeth A. Shlom, PharmD, BCPS Senior Vice President & Director Clinical Pharmacy Program | Acurity, Inc. Privileged and Confidential April 10, 2019 Privileged and Confidential Program Objectives By the end of the presentation, the pharmacist or pharmacy technician participant will be able to: ▪ Identify orphan drugs and first-in-class medications approved by the FDA in 2018. ▪ List five new drugs and their indications. ▪ Identify the place in therapy for three novel monoclonal antibodies. ▪ Discuss at least two new medications that address public health concerns. Dr. Shlom does not have any conflicts of interest in regard to this presentation. Both trade names and generic names will be discussed throughout the presentation Privileged and Confidential 2018 NDA Approvals (NMEs/BLAs) ▪ Lutathera (lutetium Lu 177 dotatate) ▪ Braftovi (encorafenib) ▪ Vizimpro (dacomitinib) ▪ Biktarvy (bictegravir, emtricitabine, ▪ TPOXX (tecovirimat) ▪ Libtayo (cemiplimab-rwic) tenofovir, ▪ Tibsovo (ivosidenib) ▪ Seysara (sarecycline) alafenamide) ▪ Krintafel (tafenoquine) ▪ Nuzyra (omadacycline) ▪ Symdeko (tezacaftor, ivacaftor) ▪ Orilissa (elagolix sodium) ▪ Revcovi (elapegademase-lvir) ▪ Erleada (apalutamide) ▪ Omegaven (fish oil triglycerides) ▪ Tegsedi (inotersen) ▪ Trogarzo (ibalizumab-uiyk) ▪ Mulpleta (lusutrombopag) ▪ Talzenna (talazoparib) ▪ Ilumya (tildrakizumab-asmn) ▪ Poteligeo (mogamulizumab-kpkc) ▪ Xofluza (baloxavir marboxil) ▪ Tavalisse (fostamatinib disodium) ▪ Onpattro (patisiran) -
Oregon Medicaid Pharmaceutical Services Prior Authorization Criteria
Oregon Medicaid Pharmaceutical Services Prior Authorization Criteria HEALTH SYSTEMS DIVISION Prior authorization (PA) criteria for fee-for-service prescriptions for Oregon Health Plan clients March 1, 2021 Contents Contents ................................................................................................................................................................ 2 Introduction........................................................................................................................................................... 7 About this guide ......................................................................................................................................... 7 How to use this guide ................................................................................................................................. 7 Administrative rules and supplemental information .................................................................................. 7 Update information............................................................................................................................................... 8 Effective March 1, 2021 ............................................................................................................................ 8 Substantive updates and new criteria ............................................................................................. 8 Clerical changes ............................................................................................................................ -
Federal Register Notice 5-1-2020 Pdf Icon[PDF – 358
Federal Register / Vol. 85, No. 85 / Friday, May 1, 2020 / Notices 25439 confidential by the respondent (5 U.S.C. schedules. Other than examination DEPARTMENT OF HEALTH AND 552(b)(4)). reports, it provides the only financial HUMAN SERVICES Current actions: The Board has data available for these corporations. temporarily revised the instructions to The Federal Reserve is solely Centers for Disease Control and the FR Y–9C report to accurately reflect responsible for authorizing, supervising, Prevention the revised definition of ‘‘savings and assigning ratings to Edges. The [CDC–2020–0046; NIOSH–233–C] deposits’’ in accordance with the Federal Reserve uses the data collected amendments to Regulation D in the on the FR 2886b to identify present and Hazardous Drugs: Draft NIOSH List of interim final rule published on April 28, potential problems and monitor and Hazardous Drugs in Healthcare 2020 (85 FR 23445). Specifically, the develop a better understanding of Settings, 2020; Procedures; and Risk Board has temporarily revised the activities within the industry. Management Information instructions on the FR Y–9C, Schedule HC–E, items 1(b), 1(c), 2(c) and glossary Legal authorization and AGENCY: Centers for Disease Control and content to remove the transfer or confidentiality: Sections 25 and 25A of Prevention, HHS. withdrawal limit. As a result of the the Federal Reserve Act authorize the ACTION: Notice and request for comment. revision, if a depository institution Federal Reserve to collect the FR 2886b chooses to suspend enforcement of the (12 U.S.C. 602, 625). The obligation to SUMMARY: The National Institute for six transfer limit on a ‘‘savings deposit,’’ report this information is mandatory.