HCPW P feedback from CHMP Presented by: Ftima Ventura (CHMP) 15 - PowerPoint PPT Presentation

HCPW P feedback from CHMP Presented by: Fátima Ventura (CHMP) 15 March 2017 An agency of the European Union

Sum m ary • CHMP opinions – New medicines (overview 2016 – Jan/ Feb 2017) – Scientific Advices/ Protocol Assistance (October 2016 – February 2017) – PRIME eligibility (October 2016 – February 2017) • HCP input provided in the context of CHMP activities 1 HCPWP feedback from CHMP – March 2017

Hum an m edicines highlights 2 0 1 6 2 HCPWP feedback from CHMP – March 2016

Hum an m edicines highlights 2 0 1 6 3 HCPWP feedback from CHMP – March 2017

Positive opinion on new active substances – Oct – Dec 1 6 Cancer Name Active S Indication EC Venclyxto Treatment of adult patients with Venclyxto chronic lymphocytic leukaemia Treatment of anaplastic lymphoma kinase (ALK)-positive advanced non- EC Alecensa alectinib small cell lung cancer (NSCLC) previously treated with crizotinib monitored (supervision) HCP EC EC decision pending EC Authorised orphan conditional marketing authorisation Restricted prescription additional monitoring 4 HCPWP feedback from CHMP – March 2017

Hum an m edicines highlights 2 0 1 6 5 HCPWP feedback from CHMP – March 2017

Positive opinion on new active substances – Oct – Dec 1 6 Rheum atology Name Active S Indication EC I nnovation Olumiant baricitinib Treatment of rheumatoid arthritis monitored (supervision) HCP EC EC decision pending EC Authorised orphan conditional marketing authorisation Restricted prescription additional monitoring 6 HCPWP feedback from CHMP – March 2017

Hum an m edicines highlights 2 0 1 6 7 HCPWP feedback from CHMP – March 2017

Positive opinion on new active substances – Oct – Dec 1 6 Haem atology Name Active S Indication EC acid lonoctocog alfa Treatment and prophylaxis of bleeding Afstyla in patients with haemophilia A 8 HCPWP feedback from CHMP – March 2017

Hum an m edicines highlights 2 0 1 6 9 HCPWP feedback from CHMP – March 2017

Positive opinion on new active substances – Oct – Dec 1 6 Hepatology/ Gastroenterology Name Active S Indication Treatment of primary biliary Ocaliva obeticholic acid cholangitis (also known as primary EC biliary cirrhosis) monitored (supervision) HCP EC EC decision pending EC Authorised orphan conditional marketing authorisation Restricted prescription additional monitoring 10 HCPWP feedback from CHMP – March 2017

Hum an m edicines highlights 2 0 1 6 11 HCPWP feedback from CHMP – March 2017

Positive opinion on new m edicines – January - February 1 7 • 4 positive recom m endations on new m edicines Rheum atology EC Name Active S Indication Xeljanz tofacitinib Treatment of rheumatoid arthritis Metabolism Name Active S Indication EC sodium zirconium Lokelma Treatment of hyperkalaemia cyclosilicate Generics, art. 58, biossimilars, hibrids, not included 12 HCPWP feedback from CHMP - March 2017

Positive opinion on new m edicines – January - February 1 7 • 4 positive recom m endations on new m edicines Endocrinology Name Active S Indication Natpar parathyroid hormone Treatment of hypoparathyroidism EC Hepatology/ Gastroenterology/ Cancer Name Active S Indication EC Prevention of nausea and vomiting in Varuby rolapitant cancer patients Generics, art. 58, biossimilars, hibrids, not included 13 HCPWP feedback from CHMP - March 2017

Scientific Advice ( October 1 6 – February 1 7 ) Drug substance type 140 Number of scientific advices 115 120 100 86 80 60 40 20 8 7 0 Chemicals Biologicals ATMP Innovative Therapeutic area 120 Number of scientific Advices 98 100 80 51 60 40 23 21 20 7 6 3 0 14 HCPWP feedback from CHMP – March 2017

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 optimise 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. 15 HCPWP feedback from CHMP – March 2017

PRI ME eligibility up to 2 3 February 2 0 1 7 16 HCPWP feedback from CHMP – March 2017

PRI ME eligibility – Oct 1 6 – Feb 2 0 1 7 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted A4250 Chemical Gastroenterology- Treatment of Progressive Familial 10-2016 Hepatology Intrahepatic Cholestasis Allogeneic Epstein- Advanced Haematology Treatment of patients with Epstein- 10-2016 Barr virus-specific therapy Barr Virus-associated Post cytotoxic T Transplant Lymphoproliferative lymphocytes (ATA129) Disorder in the allogeneic hematopoietic cell transplant setting who have failed on rituximab. MBX-8025 Chemical Gastroenterology- Treatment of Primary Biliary 10-2016 Hepatology Cholangitis Allopregnanolone Chemical Psychiatry Treatment of Postpartum 11-2016 (SAGE-547) depression Autologous CD4+ and Advanced Oncology Treatment of relapsed/refractory 12-2016 CD8+ T cells therapy diffuse large B-cell lymphoma Expressing a CD19- (DLBCL) Specific Chimeric Antigen Receptor 17 HCPWP feedback from CHMP – September 2016 (JCAR017)

PRI ME eligibility – Oct 1 6 – Feb 2 0 1 7 Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Adeno-associated viral Advanced Haematology Treatment of haemophilia A 01-2017 vector serotype 5 therapy containing a B-domain deleted variant of human coagulation factor VIII gene (BMN 270) Adeno-associated viral Advanced Neurology Treatment of paediatric patients 01-2017 vector serotype 9 therapy diagnosed with spinal muscular containing the human atrophy Type 1 SMN gene (AVXS-101) Adeno-associated viral Advanced Haematology Treatment of haemophilia B 02-2017 vector containing therapy factor IX gene variant (PF-06838435/SPK- 9001) Givosiran Chemical Endocrinology- Prevention of acute attacks of 02-2017 Gynaecology-Fertility- hepatic porphyria Metabolism HCPWP feedback from CHMP – March 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 ( Xeljanz, Lokelm a) 19 HCPWP feedback from CHMP – March 2017