Bedfordshire, Luton and Milton Keynes CCG High Cost Drugs
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Optumrx Brand Pipeline Forecast
RxOutlook® 1st Quarter 2019 OptumRx brand pipeline forecast Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug 2019 Possible launch date Ophthalmological DS-300 DS-300 Eton undisclosed SC Filed NDA 2019 unknown N disease anti-sclerostin Evenity romosozumab Amgen Osteoporosis SC Filed NDA 2/2019 Y N monoclonal antibody tetrahydrofolate iclaprim iclaprim Motif Bio Bacterial infections IV Filed NDA 2/13/2019 Y Y dehydrogenase inhibitor tazarotene/ IDP-118 Valeant retinoid/ corticosteroid Psoriasis TOP Filed NDA 2/15/2019 N N halobetasol adenosine deaminase Mavenclad cladribine Merck/ Teva resistant Multiple sclerosis PO Filed NDA 2/15/2019 Y N deoxyadenosine analog Lotemax Gel loteprednol Valeant corticosteroid Ocular inflammation OP Filed NDA 2/25/2019 N N Nex Gen etabonate turoctocog alfa glyco-PEGylated factor NN-7088 Novo Nordisk Hemophilia IV/SC Filed BLA 2/27/2019 Y N pegol VIII derivative selective sphingosine-1 BAF-312 siponimod Novartis phosphate receptor Multiple sclerosis PO Filed NDA 3/1/2019 Y N agonist midazolam midazolam UCB benzodiazepine Seizures Intranasal Filed NDA 3/1/2019 N Y (USL-261) XeriSol glucagon Xeris glucagon analog Diabetes mellitus SC Filed NDA 3/1/2019 N N Glucagon optum.com/optumrx 1 RxOutlook® 1st Quarter 2019 Route of Regulatory Estimated Specialty Orphan Drug name Generic name Company Drug class Therapeutic use administration status release date drug drug dopamine receptor JZP-507 sodium oxybate Jazz Narcolepsy -
Safety and Tolerability of Arimoclomol in Patients with Sporadic Inclusion Body Myositis: a Randomised, Double-Blind, Placebo-Controlled, Proof-Of-Concept Trial
Safety and tolerability of arimoclomol in patients with sporadic inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept trial Sporadic inclusion body myositis (IBM) is the commonest idiopathic inflammatory myopathy (IIM) occurring in patients over the age of 50 years.1-4 The prevalence of IBM differs between different populations and ethnic groups, with estimates between 4.9-70.6 per million population.5-10 IBM is distinguished from the other IIM by early asymmetric finger flexor and knee extensor weakness (leading to loss of hand function and propensity to fall) and resistance to immunosuppressive therapy. Any skeletal muscle can be affected, including oesophageal and pharyngeal muscles. Late-stage disease can be characterized by very significant morbidity including disability and loss of quality of life. Death in IBM is related to malnutrition, cachexia, aspiration, respiratory infection and respiratory failure, as a consequence of dysphagia, severe global weakness and weakness of the respiratory muscles.1-4 In a Dutch cohort, end-of-life care interventions were used by 13% of patients with IBM, highlighting the morbidity experienced by these patients and the need of supportive and palliative care in this disease.2 The aetiopathogenesis of IBM remains uncertain. The varied pathological findings observed have driven a number of theories including viral infection, accumulation of toxic proteins, autoimmune attack, myonuclear degeneration, endoplasmic reticulum stress and impairment of autophagy and proteasomal proteolysis.11-15 Muscle biopsies from patients with IBM typically show several different pathological features broadly described as inflammatory or degenerative. Inflammatory features include endomysial infiltration by mononuclear cells (predominantly CD8+ T- cells), which surround and invade otherwise normal appearing muscle fibres, and overexpression of major histocompatibility complex (MHC) class I, which is not constitutively expressed by skeletal muscle. -
International Blood/Plasma News© Is Protected by Copyright Law
EDITOR: KEITH BERMAN PUBLISHER: PATRICK ROBERT ISSN 0742-7719 Published by: The Marketing Research Bureau, Inc. 284 Racebrook Rd. Orange, CT 06477 VOLUME 35 ISSUE 12 JULY 2018 BUSINESS BRIEFS 166 BLOOD & BIOTECHNOLOGY 171 RESEARCH & DEVELOPMENT 173 PRODUCT SAFETY UPDATE 174 PEOPLE 175 NEW PRODUCTS 176 RECENT U.S. PATENTS 177 MEETINGS / SUBSCRIPTION FORM 180 ____________________COMPANIES IN THIS ISSUE__________________ ACCELERON PHARMA GRIFOLS ADMA BIOLOGICS HAEMONETICS ALKAHEST HARPOON THERAPEUTICS AMAG PHARMACEUTICALS HEMARUS THERAPEUTICS ARSANIS BIOSCIENCES GmbH ICER ARTHREX INSERM BAYER HEALTHCARE JEFFREY MODELL FOUNDATION BIO PRODUCTS LABORATORY NOVO NORDISK BIOPROTEIN TECHNOLOGIES OCTAPHARMA BIOVERATIV PHARMING CCRE HOLDINGS PLATOD CELGENE REGEN LABS SA CERUS ROCHE CHINA BIOLOGICS RUBIUS THERAPEUTICS CITIC CAPITAL HOLDINGS SANGAMO THERAPEUTICS CORD BLOOD REGISTRY SANQUIN CORD:USE CORD BLOOD BANK SHIRE CRISPR THERAPEUTICS SHIRE SINGAPORE CRTS des Armées SK CHEMICALS CRYO-CELL INTERNATIONAL TERUMO BCT CSL BEHRING VERTEX PHARMACEUTICALS DOVA PHARMACEUTICALS VFMCRP GI PARTNERS VIFOR PHARMA Notice: International Blood/Plasma News© is Protected by Copyright Law. Reproduction or Photocopy of Any Part Without The Publisher‘s Permission is Prohibited by Law. Page 166 JULY 2018 international blood/plasma news BUSINESS BRIEFS * Reflecting strong global demand for human polyvalent immunoglobulin products, HAEMONETICS reported 10.6% growth in fourth quarter revenues for its Plasma busi- ness unit, to $111.6 million from $100.9 million in the same period in 2017. Plasma business revenues for the full fiscal year ended March 31, 2018 increased by 6.7%, or by 7.1% on a con- stant currency basis. Specifically for the North American market, Plasma business revenue growth was 14.2% in the fourth quarter and 8.5% for fiscal year 2018. -
Pioneering New Markets Changing the Standard of Care
ANNUAL 2020 REPORT Pioneering New Markets Changing the Standard of Care UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 □ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or other jurisdiction of (IRS Employer incorporation or organization) Identification No.) 2855 Gazelle Court, Carlsbad, CA 92010 (Address of Principal Executive Offices) (Zip Code) 760-931-9200 (Registrant’s telephone number, including area code) Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading symbol Name of each exchange on which registered Common Stock, $.001 Par Value ‘‘IONS’’ The Nasdaq Stock Market LLC Securities registered pursuant to Section 12(g) of the Act: None Indicate by check mark if the Registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☒ No □ Indicate by check if the Registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes □ No ☒ Indicate by check mark whether the Registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the Registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. -
Vonicog Alfa
STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL USAN VONICOG ALFA PRONUNCIATION voe' ni kog al' fa THERAPEUTIC CLAIM Prophylactic treatment of bleeding episodes and Von Willebrand disease CHEMICAL NAMES 1. Blood-coagulation factor VIII, von Willebrand's 2. [618-threonine,709-aspartic acid]von Willebrand factor Homo sapiens (1381A>T,1472H>D variant) STRUCTURAL FORMULA Monomer / Monomère / Monómero SLSCRPPMVK LVCPADNLRA EGLECTKTCQ NYDLECMSMG CVSGCLCPPG 50 MVRHENRCVA LERCPCFHQG KEYAPGETVK IGCNTCVCRD RKWNCTDHVC 100 DATCSTIGMA HYLTFDGLKY LFPGECQYVL VQDYCGSNPG TFRILVGNKG 150 CSHPSVKCKK RVTILVEGGE IELFDGEVNV KRPMKDETHF EVVESGRYII 200 LLLGKALSVV WDRHLSISVV LKQTYQEKVC GLCGNFDGIQ NNDLTSSNLQ 250 VEEDPVDFGN SWKVSSQCAD TRKVPLDSSP ATCHNNIMKQ TMVDSSCRIL 300 TSDVFQDCNK LVDPEPYLDV CIYDTCSCES IGDCACFCDT IAAYAHVCAQ 350 HGKVVTWRTA TLCPQSCEER NLRENGYECE WRYNSCAPAC QVTCQHPEPL 400 ACPVQCVEGC HAHCPPGKIL DELLQTCVDP EDCPVCEVAG RRFASGKKVT 450 LNPSDPEHCQ ICHCDVVNLT CEACQEPGGL VVPPTDAPVS PTTLYVEDIS 500 EPPLHDFYCS RLLDLVFLLD GSSRLSEAEF EVLKAFVVDM MERLRISQKW 550 VRVAVVEYHD GSHAYIGLKD RKRPSELRRI ASQVKYAGSQ VASTSEVLKY 600 TLFQIFSKID RPEASRITLL LMASQEPQRM SRNFVRYVQG LKKKKVIVIP 650 VGIGPHANLK QIRLIEKQAP ENKAFVLSSV DELEQQRDEI VSYLCDLAPE 700 APPPTLPPDM AQVTVGPGLL GVSTLGPKRN SMVLDVAFVL EGSDKIGEAD 750 FNRSKEFMEE VIQRMDVGQD SIHVTVLQYS YMVTVEYPFS EAQSKGDILQ 800 RVREIRYQGG NRTNTGLALR YLSDHSFLVS QGDREQAPNL VYMVTGNPAS 850 DEIKRLPGDI QVVPIGVGPN ANVQELERIG WPNAPILIQD FETLPREAPD 900 LVLQRCCSGE GLQIPTLSPA PDCSQPLDVI LLLDGSSSFP ASYFDEMKSF 950 AKAFISKANI GPRLTQVSVL QYGSITTIDV -
Horizon Scanning Status Report June 2019
Statement of Funding and Purpose This report incorporates data collected during implementation of the Patient-Centered Outcomes Research Institute (PCORI) Health Care Horizon Scanning System, operated by ECRI Institute under contract to PCORI, Washington, DC (Contract No. MSA-HORIZSCAN-ECRI-ENG- 2018.7.12). The findings and conclusions in this document are those of the authors, who are responsible for its content. No statement in this report should be construed as an official position of PCORI. An intervention that potentially meets inclusion criteria might not appear in this report simply because the horizon scanning system has not yet detected it or it does not yet meet inclusion criteria outlined in the PCORI Health Care Horizon Scanning System: Horizon Scanning Protocol and Operations Manual. Inclusion or absence of interventions in the horizon scanning reports will change over time as new information is collected; therefore, inclusion or absence should not be construed as either an endorsement or rejection of specific interventions. A representative from PCORI served as a contracting officer’s technical representative and provided input during the implementation of the horizon scanning system. PCORI does not directly participate in horizon scanning or assessing leads or topics and did not provide opinions regarding potential impact of interventions. Financial Disclosure Statement None of the individuals compiling this information have any affiliations or financial involvement that conflicts with the material presented in this report. Public Domain Notice This document is in the public domain and may be used and reprinted without special permission. Citation of the source is appreciated. 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. -
A Stratified Approach for Cushing's Syndrome Diagnosis
Global Journal of Health Science; Vol. 11, No. 10; 2019 ISSN 1916-9736 E-ISSN 1916-9744 Published by Canadian Center of Science and Education A Stratified Approach for Cushing’s Syndrome Diagnosis Mervin Chavez1, Joselyn Rojas1,2, Miguel Aguirre1, Marjorie Villalobos1, Juan Salazar1, Luis Bello1, Roberto Añez1, Luis Olivar1, María Calvo1, Yaneth Herazo-Beltrán3, Maricarmen Chacín González3, Jhoalmis Sierra-Castrillo4 & Valmore Bermúdez-Pirela1,3 1 Endocrine and Metabolic Diseases Research Center, “Dr. Félix Gómez,” School of Medicine. University of Zulia, Maracaibo 4004, Venezuela 2 Pulmonary and Critical Care Medicine Department, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA 3 Universidad Simón Bolívar, Facultad de Ciencias de la Salud, Barranquilla, Colombia 4 Universidad de Santander (UDES), Programa de Bacteriología y Laboratorio Clínico, Cúcuta, Colombia Correspondence: Valmore Bermúdez-Pirela, MD, MgS, MPH, PhD. Universidad Simón Bolívar, Facultad de Ciencias de la Salud, Barranquilla, Colombia. E-mail: [email protected] Received: May 2, 2019 Accepted: July 3 18, 2019 Online Published: August 15, 2019 doi:10.5539/gjhs.v11n10p55 URL: https://doi.org/10.5539/gjhs.v11n10p55 Abstract Cushing’s syndrome is an endocrine disorder broadly renowned as a diagnostic challenge. From the initial clinical presentation up to the identification of the underlying etiology, it is necessary to adhere to a logical and stratified plan of action, directed to the correlation of signs and symptoms to the physiopathology of the syndrome, in order to accurately establish a diagnosis and adequate treatment. From stages as early as the patient’s first clinical evaluation, the physician should be specially attentive of a constellation of clinical signs which strongly suggest the diagnosis of Cushing’s syndrome, such as the presence of a “moon face”, a “buffalo hump”, cutaneous atrophy, proximal muscle weakness and purplish cutaneous striae, among others. -
Update on Medical Treatment for Cushing's Disease
Cuevas-Ramos et al. Clinical Diabetes and Endocrinology (2016) 2:16 DOI 10.1186/s40842-016-0033-9 REVIEWARTICLE Open Access Update on medical treatment for Cushing’s disease Daniel Cuevas-Ramos1, Dawn Shao Ting Lim2 and Maria Fleseriu2* Abstract Cushing’s disease (CD) is the most common cause of endogenous Cushing’s syndrome (CS). The goal of treatment is to rapidly control cortisol excess and achieve long-term remission, to reverse the clinical features and reduce long-term complications associated with increased mortality. While pituitary surgery remains first line therapy, pituitary radiotherapy and bilateral adrenalectomy have traditionally been seen as second-line therapies for persistent hypercortisolism. Medical therapy is now recognized to play a key role in the control of cortisol excess. In this review, all currently available medical therapies are summarized, and novel medical therapies in phase 3 clinical trials, such as osilodrostat and levoketoconazole are discussed, with an emphasis on indications, efficacy and safety. Emerging data suggests increased efficacy and better tolerability with these novel therapies and combination treatment strategies, and potentially increases the therapeutic options for treatment of CD. New insights into the pathophysiology of CD are highlighted, along with potential therapeutic applications. Future treatments on the horizon such as R-roscovitine, retinoic acid, epidermal growth factor receptor inhibitors and somatostatin-dopamine chimeric compounds are also described, with a focus on potential -
Justification
Justification to the Resolution of the Federal Joint Committee (G-BA) on an Amendment of the Pharmaceuticals Directive (AM ‑ RL): Annex XII – Resolutions on the Benefit Assessment of Medicinal Products with New Active Ingredients in Accordance with Section 35a SGB V Damoctocog alfa pegol From 20 June 2019 Contents 1. Legal basis ................................................................................................................ 2 2. Key points of the resolution ..................................................................................... 2 2.1 Additional benefit of the medicinal product in relation to the appropriate comparator therapy ..................................................................................................... 3 2.1.1 Approved therapeutic indication of damoctocog alfa pegol (Jivi®) in accordance with the summary of product characteristics ............................................ 3 2.1.2 Appropriate comparator therapy ................................................................... 3 2.1.3 Extent and probability of the additional benefit .............................................. 5 2.1.4 Summary of the assessment ........................................................................ 6 2.2 Number of patients or demarcation of patient groups eligible for treatment ...... 6 2.3 Requirements for a quality-assured application ................................................ 7 2.4 Treatment costs .............................................................................................. -
Vonicog Alfa for Severe Von Willebrand Disease
Horizon Scanning Research February 2016 & Intelligence Centre Vonicog alfa for severe von Willebrand disease LAY SUMMARY People with von Willebrand disease either have a deficiency of a blood protein called von Willebrand factor, or this protein doesn't work properly. This means that their blood cannot clot properly and they are prone to prolonged or excessive bleeding. There are three different types of von Willebrand disease – types 1, 2, and 3. Type 3 is rare and This briefing is people with this type have very low levels of von Willebrand factor in based on their blood, or none at all. information available at the time of research and a Vonicog alfa is a new drug that is given in a drip (directly into a vein). It limited literature is being studied to see whether it reduces bleeding in people with search. It is not severe von Willebrand disease, and that it is safe to use. If vonicog intended to be a alfa is licensed for use in the UK, it could provide a new treatment for definitive statement people with severe von Willebrand disease. on the safety, efficacy or effectiveness of the NIHR HSRIC ID: 5818 health technology covered and should not be used for commercial purposes or commissioning without additional information. This briefing presents independent research funded by the National Institute for Health Research (NIHR). The views expressed are those of the author and not necessarily those of the NHS, the NIHR or the Department of Health. NIHR Horizon Scanning Research & Intelligence Centre, University of Birmingham. Email: [email protected] Web: www.hsric.nihr.ac.uk Horizon Scanning Research & Intelligence Centre TARGET GROUP Von Willebrand disease (VWD): severe – first line. -
Rxoutlook® 1St Quarter 2019
® RxOutlook 1st Quarter 2020 optum.com/optumrx a RxOutlook 1st Quarter 2020 Orphan drugs continue to feature prominently in the drug development pipeline In 1983 the Orphan Drug Act was signed into law. Thirty seven years later, what was initially envisioned as a minor category of drugs has become a major part of the drug development pipeline. The Orphan Drug Act was passed by the United States Congress in 1983 in order to spur drug development for rare conditions with high unmet need. The legislation provided financial incentives to manufacturers if they could demonstrate that the target population for their drug consisted of fewer than 200,000 persons in the United States, or that there was no reasonable expectation that commercial sales would be sufficient to recoup the developmental costs associated with the drug. These “Orphan Drug” approvals have become increasingly common over the last two decades. In 2000, two of the 27 (7%) new drugs approved by the FDA had Orphan Designation, whereas in 2019, 20 of the 48 new drugs (42%) approved by the FDA had Orphan Designation. Since the passage of the Orphan Drug Act, 37 years ago, additional regulations and FDA designations have been implemented in an attempt to further expedite drug development for certain serious and life threatening conditions. Drugs with a Fast Track designation can use Phase 2 clinical trials to support FDA approval. Drugs with Breakthrough Therapy designation can use alternative clinical trial designs instead of the traditional randomized, double-blind, placebo-controlled trial. Additionally, drugs may be approved via the Accelerated Approval pathway using surrogate endpoints in clinical trials rather than clinical outcomes. -
Patent Application Publication ( 10 ) Pub . No . : US 2019 / 0192440 A1
US 20190192440A1 (19 ) United States (12 ) Patent Application Publication ( 10) Pub . No. : US 2019 /0192440 A1 LI (43 ) Pub . Date : Jun . 27 , 2019 ( 54 ) ORAL DRUG DOSAGE FORM COMPRISING Publication Classification DRUG IN THE FORM OF NANOPARTICLES (51 ) Int . CI. A61K 9 / 20 (2006 .01 ) ( 71 ) Applicant: Triastek , Inc. , Nanjing ( CN ) A61K 9 /00 ( 2006 . 01) A61K 31/ 192 ( 2006 .01 ) (72 ) Inventor : Xiaoling LI , Dublin , CA (US ) A61K 9 / 24 ( 2006 .01 ) ( 52 ) U . S . CI. ( 21 ) Appl. No. : 16 /289 ,499 CPC . .. .. A61K 9 /2031 (2013 . 01 ) ; A61K 9 /0065 ( 22 ) Filed : Feb . 28 , 2019 (2013 .01 ) ; A61K 9 / 209 ( 2013 .01 ) ; A61K 9 /2027 ( 2013 .01 ) ; A61K 31/ 192 ( 2013. 01 ) ; Related U . S . Application Data A61K 9 /2072 ( 2013 .01 ) (63 ) Continuation of application No. 16 /028 ,305 , filed on Jul. 5 , 2018 , now Pat . No . 10 , 258 ,575 , which is a (57 ) ABSTRACT continuation of application No . 15 / 173 ,596 , filed on The present disclosure provides a stable solid pharmaceuti Jun . 3 , 2016 . cal dosage form for oral administration . The dosage form (60 ) Provisional application No . 62 /313 ,092 , filed on Mar. includes a substrate that forms at least one compartment and 24 , 2016 , provisional application No . 62 / 296 , 087 , a drug content loaded into the compartment. The dosage filed on Feb . 17 , 2016 , provisional application No . form is so designed that the active pharmaceutical ingredient 62 / 170, 645 , filed on Jun . 3 , 2015 . of the drug content is released in a controlled manner. Patent Application Publication Jun . 27 , 2019 Sheet 1 of 20 US 2019 /0192440 A1 FIG .