HCPW P feedback from CHMP Presented by: Fátima Ventura (CHMP) 26 September 2018 An agency of the European Union
Sum m ary • CHMP opinions (Apr 2018 – Sep 2018) – New medicines – Other issues related to authorised medicines – Scientific Advices/ Protocol Assistance – PRIME eligibility (Apr 2018 – Jul 2018) 1 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Alim entary tract & Metabolism ( 2 ) Nam e DS I ndication Mepsevii vestronidase alfa Mucopolysaccharidosis type VII EC SO – Results from a Disease Monitory Program - long-term on effectiveness and safety and characterize MPS VII (clinical, progression, history) Myalepta metreleptin Leptin deficiency EC (lipodystrophy) EP/ EM – HCP and Patient/ Carer EM 2 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Blood ( 3 ) Nam e DS I ndication Cablivi caplacizumab Acquired thrombotic EC thrombocytopenic EP/ EM – Patient alert card Veyvondi vonicog alfa von Willebrand disease EC Jivi damoctocog alfa haemophilia A (congenital EC pegol factor VIII deficiency) 3 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Antiinfective drugs ( 5 ) Nam e DS I ndication Vabom ere meropenem / Complicated urinary tract EC vaborbactam infection (cUTI), complicated intra-abdominal infection (cIAI), hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP) Xerava eravacycline complicated intra-abdominal EC infections in adults 4 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Antiinfective drugs Nam e DS I ndication Biktarvy bictegravir / HIV-1 infection EC emtricitabine / tenofovir alafenamide Delstrigo doravirine / HIV-1 infection lamivudine / EC tenofovir disoproxil Pifeltro doravirine HIV-1 infection EC 5 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Cancer ( 1 1 ) Nam e DS I ndication Apealea paclitaxel Ovarian cancer EC Alunbrig brigatinib anaplastic lymphoma kinase positive advanced non-small cell lung cancer previously EC treated with crizotinib Braftovi encorafenib In combination with binimetinib is indicated for the treatment of adult patients with EC unresectable or metastatic melanoma with a BRAF V600 mutation I m finzi durvalumab Non-small cell lung cancer EC Kym riah durvalutisagenlecl Acute lymphoblastic leukaemia eucelmab (ALL) and Diffuse large B-cell EC lymphoma (DLBCL) ATMP; EP/ EM – HCP + Patients (Patient alert card); 1 st CAR-T cells approved EU 6 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Cancer Nam e DS I ndication Mektovi binimetinib In combination with encorafenib is indicated for the EC treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation Nerlynx neratinib breast cancer EC EP/ EM – HCP + Patients EM Verzenios abemaciclib locally advanced or metastatic EC breast cancer Vyxeos daunorubicin / acute myeloid leukaemia EC cytarabine 7 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Cancer Nam e m ogam ulizum ab I ndication Poteligeo mogamulizumab Mycosis fungoides or Sézary EC syndrome Yescarta axicabtagene diffuse large cell lymphoma ciloleucel (DLBCL) and primary EC mediastinal B-cell lymphoma (PMBCL) ATMP; EP/ EM – HCP + Patients (Patient alert card); 1 st CAR-T cells approved 8 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 I m m unom odulation Nam e DS I ndication I lum etri tildrakizumab moderate to severe plaque EC psoriasis Respiratory system Nam e DS I ndication Sym kevi tezacaftor / cystic fibrosis EC ivacaftor Genetic Disorders Nam e DS I ndication Luxturna voretigene retinal dystrophies caused by EC neparvovec RPE65 mutations ATMP; Gene Therapy 9 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Nervous system ( 6 ) Nam e DS I ndication Aim ovig erenumab Prophylaxis of migraine EC 1 st monoclonal antibody therapy for prevention of migraine Em gality galcanezumab Prophylaxis of migraine EC New medicine for the prevention of migraine EC Rxulti brexpiprazole Schizophrenia Slenyto melatonin insomnia in children and EC PUMA adolescents with autism spectrum disorder or Smith- Magenis syndrome 10 HCPWP feedback from CHMP – September 2018
New m edicines – Apr – Sep 2 0 1 8 Nervous system Nam e DS I ndication Onpattro patisiran hereditary transthyretin- EC mediated amyloidosis Tegsedi inotersen Hereditary transthyretin EC amyloidosis Metabolism Nam e DS I ndication Duzallo lesinurad / Hyperuricaemia in gout EC allopurinol patients 11 HCPWP feedback from CHMP – September 2018
Other issues related to authorised m edicines Changes in the Product I nform ation Nam e DS I ndication Change Viekirax Ombitasvir / Hepatitis C Contra-indication paritaprevir / Patients with moderate to ritonavir severe hepatic impairment (Child-Pugh B) Xofigo radium-223 Restriction of indication Cancer of the prostate dichloride To be used only after two previous treatments or when other treatments cannot be taken Referral – Art. 2 0 – PRAC/ CHMP/ EC 12 HCPWP feedback from CHMP – September 2018
Other issues related to authorised m edicines Referral Art. 3 1 - Valsartan o Valsartan: EMA is currently reviewing medicines containing valsartan from Zhejiang Huahai Pharmaceuticals (Linhai, China) following detection of an impurity o N-nitrosodimethylamine (NDMA) - probable human carcinogen (results from laboratory tests) o Presence of NDMA was unexpected and possible related to changes in the active substance manufacturing. NCAs are recalling medicines containing valsartan supplied by Zhejiang Huahai. 13 HCPWP feedback from CHMP – September 2018
Other issues related to authorised m edicines Referral Art. 3 1 - Valsartan o Valsartan: EMA is now evaluating potential impact on patients of the exposure to NDMA in valsartan medicines. Review will also consider whether other valsartan medicines may be affected. Information for patients (not stop treatment)/ HCP o + Losartan, candesartan, irbesartan, and olm esartan: Low levels of N-nitrosodiethylamine (NDEA) present in one batch of Losartan from Hetero Labs, India Like valsartan – tetrazole ring – potential NDMA EMA is expanding review of valsartan for NDEA – no immediate risk (low levels NDEA) 14 HCPWP feedback from CHMP – September 2018
Scientific Advice/ Protocol assis. ( Apr 1 8 – Sep 1 8 ) Drug substance type 94 100 80 67 Number SA 60 40 13 20 6 3 0 Chemicals Biologicals ATMP Innovative HTA Therapeutic area 60 52 50 50 Number SA 35 40 30 17 15 20 8 10 2 2 0 15 HCPWP feedback from CHMP – April 2018
PRI ME eligibility – Abr – Sep 2 0 1 8 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Adeno-associated viral Advanced Other Treatment of X-linked myotubular 05-2018 vector serotype 8 Therapy myopathy containing the human MTM1 gene (AT132) Allogeneic umbilical Advanced Immunology- Treatment in Haematopoietic 05-2018 cord blood CD34+ cells Therapy Rheumatology- Stem Cell Transplantation (HSCT) cultured ex vivo with Transplantation Notch ligand Delta1 (NLA101) Autologous T cells Advanced Oncology Treatment of adult patients with 05-2018 transduced with Therapy relapsed or refractory mantle cell retroviral vector lymphoma encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (KTE-C19) MV-CHIK vaccine Biological Vaccines Prevention of Chikungunya fever 05-2018 Deoxycytidine / Chemical Endocrinology- Treatment of Thymidine Kinase 2 06-2018 deoxythymidine Gynaecology-Fertility- Deficiency Metabolism HCPWP feedback from CHMP – April 2018 16
PRI ME eligibility – Apr – Jul 2 0 1 8 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Mycobacterium Immunological Vaccines Active immunization against 06-2018 tuberculosis tuberculosis disease in newborns, (MTBVAC) adolescents and adults Setmelanotide Chemical Endocrinology- Treatment of obesity and the 06-2018 Gynaecology-Fertility- control of hunger associated with Metabolism deficiency disorders of the MC4R receptor pathway Chimeric 2'-O-(2- Chemical Neurology Treatment of Huntington’s Disease 07-2018 methoxyethyl) modified oligonucleotide targeted to huntingtin RNA (RO7234292) Autologous Advanced Neurology Treatment of cerebral 07-2018 haematopoeitic stem therapy adrenoleukodystrophy cells transduced with lentiviral vector Lenti- D encoding the human ATP-binding cassette, sub-family D (ALD), member 1 (ABCD1) from cDNA HCPWP feedback from CHMP – April 2018 17
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