CHMP feedback to working parties Presented by: Fátima Ventura (CHMP) 25 September 2019 An agency of the European Union
Sum m ary (September 2018 – September 2019) • CHMP opinions – New medicines – Community Reviews – PRIME eligibility (September 2018 – July 2019) • HCP/ Patients input provided in the context of CHMP activities CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Accelerated assessment monitored (supervision) HCP EC EC EC decision pending Authorised orphan conditional marketing authorisation Restricted prescription additional monitoring Exceptional circumstances CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Haem atology Name Active S Indication EC Treatment of direct factor Xa(FXa) inhibitor (apixaban or rivaroxaban) Ondexxya (Feb/19) andexanet alfa when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding Press release: First antidote for reversal of anticoagulation w ith factor Xa inhibitors apixaban and rivaroxaban Name Active S Indication autologous CD34+ cells Treatment of transfusion-dependent Zynteglo (Mar/19) EC encoding βA -T87Q- β -thalassaemia globin gene Advanced Therapy Press release: New gene therapy to treat rare inherited blood condition CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Haem atology Name Active S Indication X EC Treatment and prophylaxis of turoctocog alfa bleeding in patients 12 years and Esperoct (Apr/19) pegol above with haemophilia A (congenital factor VIII deficiency) Cancer Name Active S Indication EC ? Treatment of patients with solid tumours that display a neurotrophic Vitrakvi (Jul/19) larotrectinib tyrosine receptor kinase (NTRK) gene fusion Press release: First “histology-independent” treatm ent for solid tum ours w ith a specific gene m utation – I novative design – basket trial CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Vaccine Name Active S Indication For the prevention of dengue caused EC by dengue virus serotypes 1, 2, 3 and dengue tetravalent 4 in people who are between 9 and Dengvaxia (Oct/19) vaccine (live, 45 years old, live in an endemic area attenuated) and already had a prior dengue virus infection Press release: First vaccine for prevention of dengue CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Metabolism Name Active S Indication EC Palynziq (Feb/19) pegvaliase Treatment of phenylketonuria Press release: CHMP recom m ends authorisation of new treatm ent for phenylketonuria, a rare inherited m etabolic disease Name Active S Indication EC Treatment of familial Waylivra (Feb/19) volanesorsen chylomicronaemia syndrome Press release: First treatm ent for rare disease characterised by high levels of triglycerides in blood CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Neurology Name Active S Indication EC Emgality (Sep/18) galcanezumab Prophylaxis of migraine Press release: New m edicine for the prevention of m igraine Name Active S Indication EC Symptomatic treatment of myotonia Namuscla (Oct/18) mexiletine hcl in adult patients with non-dystrophic myotonic disorders Press release: First treatm ent for rare inherited m uscle contraction disorders CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Neurology Name Active S Indication EC substitution treatment for opioid Sixmo (Apr/19) buprenorphine drug dependence I m plant Name Active S Indication EC Treatment of seizures associated with Epidyolex (Jul/19) cannabidiol Lennox-Gastaut syndrome or Dravet syndrome CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 I nfectious Diseases Name Active S Indication EC Treatment of complicated and Dectova (Feb/19) zanamivir potentially life-threatening influenza Ophthalm ology Name Active S Indication EC voretigene Treatment of retinal dystrophies caused Luxturna (Sep/18) neparvovec by RPE65 mutations Press release: New gene therapy for rare inherited disorder causing vision loss recom m ended for approval CHMP feedback to working parties, 25 September 2019
Positive opinion – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Other areas Name Active S Indication Art. 5 8 / W HO Treatment of human African Fexinidazole Winthrop fexinidazole trypanosomiasis (HAT) due to (Nov/18) Trypanosoma brucei gambiense Press release: CHMP recom m ends first oral-only treatm ent for sleeping sickness CHMP feedback to working parties, 25 September 2019
Negative opinions – Septem ber 2 0 1 8 – Septem ber 2 0 1 9 Neurology EC Name Active S Indication Treatment of Duchenne muscular Exondys (Set/18) eteplirsen dystrophy Cancer Name Active S Indication EC Cabazitaxel Teva (Apr/19) cabazitaxel Treatment of prostate cancer CHMP feedback to working parties, 25 September 2019
Com m unity review s ( Art. 3 1 ) Sep2 0 1 8 – Sep 2 0 1 9 ) Object Scope Sep/18 Sartan medicines ( Angiotensin -II- Started with Valsartan – extended to other (Start Jul/18) receptor antagonists) “Sartans” - Review of impurities (N- Nitrosodimethylamine (NDMA)) Dec/2018 (Start) Fosfomycin -containing medicinal DE - Significant differences between Member products (Antibiotics) States - reappraisal of the role of fosfomycin in the context of increasing resistance to antibiotics Dec/2018 (opinion) Metamizole -containing medicinal PL - Substantial differences between Member products (analgesic) States - aligning doses and use during pregnancy and breastfeeding Dec/2018 Omega-3 fatty acid medicines SE – Efficacy - not effective in preventing further (opinion)/Mar/2019 heart problems after a heart attack (opinion re-exam) Jan/2019 Direct oral anticoagulants Eliquis Reviewing the results of a study - risk of major (apixaban), Pradaxa (dabigatran bleedings vs other anticoagulants etexilate) and Xarelto (rivaroxaban) CHMP feedback to working parties, 25 September 2019
Com m unity review s ( Art. 3 1 / 2 0 ) Sep 2 0 1 8 – Sep 2 0 1 9 ) Object Scope Apr/19 Lartruvo ( olaratumab ) with Results of the ANNOUNCE study (SO - CMA) - not better doxorubicin than monotherapy with doxorubicin in increase survival of patients with soft tissue cancer – MA revocation May/2019 (Start) Methocarbamol and DE – assessment of B/R due to lack of efficacy - short- paracetamol -containing term, symptomatic treatment of painful muscle spasms medicines (analgesic) associated with acute musculoskeletal disorders, and potential interactions between active ingredients Jun/2019 (opinion) Bacterial lysates -containing IT - should only be used for the prevention of recurrent medicinal products indicated for respiratory infections, with the exception of pneumonia respiratory conditions - no robust data on efficacy at treating existing respiratory infections, or prevention of pneumonia Sept/2019 (start) Ranitidine -containing medicinal B/R due to concerns on preliminary results showing the products presence of N-Nitrosodimethylamine (NDMA) in some batches and preliminary findings that NDMA could be generated under certain conditions in vivo CHMP feedback to working parties, 25 September 2019
PRI ME eligibility Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Lonafarnib Chemical Infectious Diseases Treatment of hepatitis D virus infection Dec/2018 Anti-respiratory syncytial Prevention of lower respiratory tract virus human IgG1 κ Biological Infectious diseases infection caused by respiratory syncytial Jan/2019 monoclonal antibody virus (MEDI889 Purified Inactivated Zika Active immunization for the prevention of Vaccines Immunological Mar/2019 Virus vaccine (TAK-426) disease caused by Zika virus Autologous human T cells Treatment of adult patients with relapsed or genetically modified ex-vivo refractory multiple myeloma, whose prior with a lentiviral vector regimens included a proteasome inhibitor, encoding a chimeric antigen Advanced therapy Oncology Mar/2019 an immunomodulatory agent and an anti- receptor (CAR) for B-cell CD38 antibody and who had disease maturation antigen (BCMA) progression on the last regime (JNJ-68284528) Genetically modified replication-incompetent Treatment of Dystrophic Epidermolysis herpes simplex virus-1 Advanced therapy Dermatology Mar/2019 Bullosa expressing collagen VII (KB103) CHMP feedback to working parties, 25 September 2019
PRI ME eligibility Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Treatment of rituximab refractory Post- Allogeneic EBV-specific Advanced Therapy Oncology Transplant Lymphoproliferative Disorder May/2019 Cytotoxic T Lymphocyte (PTLD) Antisense oligonucleotide complementary to the exonic Treatment of Leber’s congenital amaurosis splicer enhancer sequence at Chemical Ophthalmology due to the p.Cys998X mutation in the Jul/2019 intron 26 of the centrosomal CEP290 Gene protein 290 pre-mRNA(QR- 110) CHMP feedback to working parties, 25 September 2019
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