HCPW P/ PCW P feedback from CHMP Presented by: Ftima Ventura - - PowerPoint PPT Presentation

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HCPW P/ PCW P feedback from CHMP Presented by: Ftima Ventura - - PowerPoint PPT Presentation

Oct 28, 2022 299 likes •506 views

HCPW P/ PCW P feedback from CHMP Presented by: Ftima Ventura (CHMP) 28 June 2017 An agency of the European Union Sum m ary CHMP opinions New medicines (March - May 2017) Scientific Advices/ Protocol Assistance (March May

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An agency of the European Union

HCPW P/ PCW P feedback from CHMP

Presented by: Fátima Ventura (CHMP) 28 June 2017

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HCPWP feedback from CHMP – June 2017 1

Sum m ary

  • CHMP opinions

– New medicines (March - May 2017) – Scientific Advices/ Protocol Assistance (March – May 2017) – PRIME eligibility (March – May 2017)

  • HCP/ Patients input provided in the context of CHMP

activities

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HCPWP feedback from CHMP – June 2017 2

Positive opinion on new active substances – March – May 2 0 1 7

Cancer

Name Active S Indication

Axumin fluciclovine (18F) Diagnostic agent for the detection of recurrence of prostate cancer with positron emission tomography (PET) imaging Besponsa inotuzumab

  • zogamicin

Treatment of acute lymphoblastic leukaemia

EC

  • rphan

conditional marketing authorisation Restricted prescription additional monitoring

EC

Authorised

EC EC decision pending

monitored (supervision) HCP

EC

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HCPWP feedback from CHMP – June 2017 3

Haem atology

Name Active S Indication Refixia nonacog beta pegol Treatment of haemophilia B

EC Positive opinion on new active substances – March – May 2 0 1 7

  • rphan

conditional marketing authorisation Restricted prescription additional monitoring

EC

Authorised

EC EC decision pending

monitored (supervision) HCP

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HCPWP feedback from CHMP – June 2017 4

Vaccine

Name Active S Indication

Trumenba meningococcal group B vaccine (recombinant, adsorbed) Prophylaxis against invasive meningococcal disease caused by meningococcal serogroup B bacteria

  • rphan

conditional marketing authorisation Restricted prescription additional monitoring

EC

Authorised

EC EC decision pending

monitored (supervision) HCP

Positive opinion on new active substances – March – May 2 0 1 7

EC

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HCPWP feedback from CHMP – June 2017 5

Neurology

Name Active S Indication

Brineura cerliponase alfa Treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease

Positive opinion on new active substances – March – May 2 0 1 7

EC

Press release: New m edicine for rare neurodegenerative disorder in children

Name Active S Indication

Spinraza nusinersen Treatment of spinal muscular atrophy

EC

Press release: First m edicine for spinal m uscular atrophy

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HCPWP feedback from CHMP – June 2017 6

Psychiatry

Name Active S Indication

Reagila cariprazine Treatment of schizophrenia

Positive opinion on new active substances – March – May 2 0 1 7

EC

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HCPWP feedback from CHMP – June 2017 7

I m m uno-system

Name Active S Indication

Kevzara sarilumab Treatment of rheumatoid arthritis Kyntheum brodalumab Treatment of moderate to severe plaque psoriasis

Positive opinion on new active substances – March – May 2 0 1 7

EC

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HCPWP feedback from CHMP – June 2017 8

Ophthalm ology

Name Active S Indication

Oxervate cenegermin Treatment of moderate to severe neurotrophic keratitis

Positive opinion on new active substances – March – May 2 0 1 7

EC EC Metabolism

Name Active S Indication

Veltassa patiromer Treatment of hyperkalaemia

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HCPWP feedback from CHMP – June 2017 9

Bone defect

Name Active S Indication

Spherox spheroids of human autologous matrix- associated chondrocytes Repair of certain cartilage defects of the knee

Positive opinion on new active substances – March – May 2 0 1 7

EC

Advanced Therapy Press release: New advanced therapy to repair cartilage defects in the knee

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HCPWP feedback from CHMP – June 2017 10

Cancer

Name Active S Indication

Adlumiz anamorelin hydrochloride Treatment of anorexia, cachexia or unintended weight loss in patients with non-small cell lung cancer

Negative opinions – March – May 2 0 1 7

  • Studies proof marginal efficacy and no effect on patients’ quality of life.
  • Following an inspection at clinical study sites - safety data had not been recorded adequately -

evaluation of potential risks with Adlumiz was not possible.

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HCPWP feedback from CHMP – June 2017 11

Cancer

Name Active S Indication

Human IgG1 monoclonal antibody specific for human interleukin-1 alpha XBiotech human IgG1 monoclonal antibody specific for human interleukin-1 alpha Treatment of advanced colorectal cancer

Negative opinions – March – May 2 0 1 7

  • Did not show clear improvements in either lean body mass or quality of life.
  • Increased risk of infection in patients taking the medicine, which was not considered acceptable

in vulnerable patients receiving palliative care.

  • Inadequate controls of the manufacturing process to ensure the medicine would have the same

quality as the product used in clinical trials.

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HCPWP feedback from CHMP – June 2017 12

Haem atology

Name Active S Indication

Masipro masitinib Treatment of systemic mastocytosis

Negative opinions – March – May 2 0 1 7

  • Questioned reliability of the study results due to serious fails observed during a routine GCP

(good clinical practice) inspection at the study sites.

  • Major changes were made to the study design while the study was ongoing, which made the

results difficult to interpret.

  • Limited safety data of the medicine with concerns regarding the adverse effects: neutropenia

(low levels of white blood cells) and harmful effects on the skin and liver, of relevance particularly because the medicine was to be used long term.

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HCPWP feedback from CHMP – June 2017 13

Scientific Advice ( January- May 2 0 1 7 )

97 72 18 7 20 40 60 80 100 120 Chemicals Biologicals ATMP Innovative

Number of scientific advices

Drug substance type

54 4 4 23 22 14 19 60

10 20 30 40 50 60 70

Number of scientific Advices

Therapeutic area

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HCPWP feedback from CHMP – June 2017 14

PRI ME

  • Enhance support for the development of medicines that target an unmet

medical need.

  • Interaction and early dialogue with developers of promising medicines, to
  • ptimise development plans and speed up evaluation so these medicines

can reach patients earlier.

  • Built on the existing regulatory framework and tools already available such

as scientific advice and accelerated assessment.

  • Improving clinical trial designs - data generated suitable for evaluating a

MAA

  • Patients only participate in trials designed to provide the data necessary for

an application - best use of limited resources.

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HCPWP feedback from CHMP – June 2017 15

PRI ME eligibility up to 1 8 May 2 0 1 7

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HCPWP feedback from CHMP – June 2017 16

PRI ME eligibility – March – May 2 0 1 7

Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Adeno-associated viral vector serotype 5 containing human factor IX gene (AMT- 060) Advanced therapy Haematology

  • Hemostaserology

Treatment of severe haemophilia B 04-2017 Asunercept Biological Oncology Treatment of glioblastoma 05-2017 Olipudase alfa Biological Endocrinology

  • Gynaecology
  • Fertility
  • Metabolism

Treatment of non-neurological manifestations of acid sphingomyelinase deficiency 05-2017 Rapastinel Chemical Psychiatry Adjunctive treatment of major depressive disorder 05-2017

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HCPWP feedback from CHMP – June 2017 17

PRI ME eligibility – March – May 2 0 1 7

Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Synthetic 47-amino- acid N-myristoylated lipopeptide, derived from the preS region

  • f hepatitis B virus

Chemical Infectious Diseases Treatment of chronic hepatitis D infection 05-2017 Recombinant IgG degrading enzyme of Streptococcus pyogenes Biological Immunology

  • Rheumatology
  • Transplantation

Prevention of graft rejection following solid

  • rgan transplantation)

05-2017

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HCPWP feedback from CHMP – June 2017 18

I nteraction betw een CHMP and HCP - Participation in Scientific Advisory Groups and ad-hoc Experts Groups

  • Contributing for decision on recom endations

Name Active S Indication Refixia nonacog beta pegol Treatment of haemophilia B Brineura cerliponase alfa Treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease Reagila cariprazine Treatment of schizophrenia

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HCPWP feedback from CHMP – June 2017 19

I nteraction betw een CHMP and Patient’s representatives

  • Participation

in CHMP plenary sessions

  • Contributing for decision on recom endations ( Adlum iz)

Name Active S Indication

Adlumiz anamorelin hydrochloride Treatment of anorexia, cachexia or unintended weight loss in patients with non-small cell lung cancer

  • Patients’ participation at the oral explanation for Adlumiz was perceived very positive by CHMP

members - patients’ view major help for CHMP’s deliberations.

  • Adlumiz was a classical benefit-risk discussion, with the possibility that Adlumiz may help (a little)

in a few patients, with quite some uncertainty on the safety side.

  • It was important for the Committee to hear from patients a clear “for this product, we do not

want to be given the choice”.