HCPW P feedback from CHMP Presented by: Fátima Ventura (CHMP) 18 April 2018 An agency of the European Union
Sum m ary • CHMP opinions (overview Oct 2017 – Mar 2018) – New medicines – Scientific Advices/ Protocol Assistance – PRIME eligibility • HCP/ P input provided in the context of CHMP activities 1 HCPWP feedback from CHMP – April 2018
Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 Nam e DS I ndication Adynovi rurioctocog alfa Haemophilia A EC pegol Ad hoc expert group ( included 2 Patients’ representatives) Post-authorisation safety study (PASS): In order to investigate potential effects - Q1 2019 Hem libra emicizumab Haemophilia A - factor VIII EC inhibitors EP/ EM - Physician, Patient/ Carer, and Laboratory professionals educational material I ntrarosa prasterone Vulvar and vaginal atrophy in EC postmenopausal women Crysvita burosumab X-linked EC hypophosphataemia Lam zede velmanase alfa non-neurological EC manifestations of mild to moderate alpha- mannosidosis Ad hoc expert group ( included 2 Patients’ representatives) Post-MA : Long-term data on effectiveness and safety data from a registry of patients + Annual reports/ annual re-assessment + Paediatric Study - Final Study report: November 2020 2 HCPWP feedback from CHMP – April 2018
Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 Nam e DS I ndication Fasenra benralizumab Severe eosinophilic asthma EC Jorveza budesonide Eosinophilic esophagitis EC Alofisel darvadstrocel Complex perianal fistulas in EC patients with Crohn’s disease EP/ EM - Guide for pharmacists with instructions on the appropriate reception and storage. Guide (video) for surgeons and other HCP involved in the preparation and administration. Guide for surgeons and other HCP describing the method of administration and providing information on potential for microbial information and advice on steps to follow in case a positive culture is identified. Relevant information on the risk of medication errors and the potential for transmission of infectious agents and details on how to minimise these, including reception, storage and administration instructions (i.e. fistula conditioning, preparation and injection). Post-MA : To follow-up on the efficacy of Alofisel, the MAH should submit the results of a Phase III randomised double-blind, placebo-controlled study investigating a single administration - 2Q/ 3Q 2022 Ocrevus ocrelizumab Relapsing forms of MS and EC primary progressive MS SAG ( included 2 Patients’ representatives) 3 HCPWP feedback from CHMP – April 2018
Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 Nam e DS I ndication Ozem pic semaglutide Type 2 diabetes EC Steglatro ertugliflozin Type 2 diabetes EC Seglurom et ertugliflozin / Type 2 diabetes EC metformin Steglujan ertugliflozin / Type 2 diabetes EC sitagliptin Am glidia glibenclamide Neonatal diabetes EC Measure to minimise medication errors - Number of presentations for the two different dose strengths and syringes EM - Visual educational material for physicians and pharmacists Alkindi Replacement therapy of adrenal hydrocortisone EC insufficiency in infants, children and adolescents 4 HCPWP feedback from CHMP – April 2018
Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 Nam e DS I ndication Prevym is letermovir Prophylaxis of CMV reactivation and disease in adult CMV- EC seropositive recipients of HSCT Post-MA : In order to optimise the sterility assurance level implement the measures concerning development, validation and introduction of terminal sterilisation. Shingrix herpes zoster Prevention of herpes zoster vaccine (HZ) and post-herpetic EC (recombinant, neuralgia (PHN), in adults > 50 adjuvanted) years Alpivab peramivir Uncomplicated influenza EC Juluca dolutegravir / HIV infection EC rilpivirine Acute myeloid leukaemia Mylotarg gemtuzumab EC ozogamicin Rubraca rucaparib Relapsed or progressive EC ovarian cancer SAG ( included 2 Patients’ representatives) 5 HCPWP feedback from CHMP – April 2018
Scientific Advice/ Protocol assis. ( Oct 1 7 – Mar 1 8 ) Drug substance type 150 117 Number SA 100 84 50 20 14 7 0 Chemicals Biologicals ATMP Innovative HTA Therapeutic area 100 78 Number SA 80 59 60 26 25 40 22 13 9 20 2 0 6 HCPWP feedback from CHMP – April 2018
PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Entrectinib Chemical Oncology Treatment of NTRK fusion- 10-2017 positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or who have no acceptable standard therapy Humanized antibody Biological Oncology Treatment of relapsed and 10-2017 targeting B cell refractory multiple myeloma maturation antigen patients whose prior therapy conjugated with included a proteasome inhibitor, maleimidocaproyl an immunomodulatory agent and monomethyl an anti-CD38 antibody auristatin F Human Biological Dermatology Treatment of X-linked 10-2017 immunoglobulin G1 hypohidrotic ectodermal dysplasia constant region - human ectodysplasin- A1 receptor-binding domain fusion protein HCPWP feedback from CHMP – April 2018 7
PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Autologous T Advanced Oncology Treatment of relapsed and 11-2017 lymphocyte-enriched therapy refractory multiple myeloma population of cells patients whose prior therapy transduced with a included a proteasome inhibitor, lentiviral vector an immunomodulatory agent and encoding a chimeric an anti-CD38 antibody antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3- zeta intracellular signalling domains (bb2121) LR12 Chemical Infectious Diseases Treatment of Septic Shock 11-2017 Recombinant Biological Other Treatment of osteogenesis 11-2017 humanised imperfecta types I, III and IV monoclonal IgG2 lambda antibody against human sclerostin (BPS804) HCPWP feedback from CHMP – April 2018 8
PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Adenovirus associated Advanced Oncology Treatment of achromatopsia 02-2018 viral vector serotype 8 therapy associated with defects in CNGB3 containing the human CNGB3 gene (AAV2/8- hCARp.hCNGB3) Lumasiran Chemical Uro-nephrology Treatment of Primary 03-20178 Hyperoxaluria Type 1 HCPWP feedback from CHMP – April 2018 9
I nteraction betw een CHMP & HCP & Patients Nam e I ndication Consultation Outcom e √ haemophilia A Ad-Hoc (2 Pts) Adynovi √ multiple sclerosis SAG (2 Pts) Ocrevus alcohol dependence Ad-Hoc X Alcover ( A-3 2 ) schizophrenia Ad-Hoc (2 Pts) X Fanaptum √ alpha-mannosidosis Ad-Hoc (2 Pts) Lamzede early breast cancer SAG (2 Pts) X Nerlynx advanced breast cancer SAG (2 Pts) X Onzeald met. colorectal cancer SAG (2 Pts) W ithdraw n Opdivo ( ext. ind.) √ prog. ovarian cancer SAG (2 Pts) Rubraca Duchenne SAG (2 Pts)/ CHMP X Raxone ( ext. ind.; after re-exm ) muscular dystrophy osteoporosis Ad-Hoc (2 Pts) X Eladynos multiple myeloma SAG (2 Pts) X Aplidin ( after re-exam ) 10 HCPWP feedback from CHMP – April 2018
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