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Shadows Cast Over JAK Class Following Xeljanz Mortality Signal

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Shadows Cast Over JAK Class Following Xeljanz Mortality Signal 1 March 2019 No. 3944 Scripscrip.pharmaintelligence.informa.com Pharma intelligence | informa to evaluate cardiovascular safety for both doses against an active comparator, as opposed to relying on post-marketing event reporting. On Feb. 19, Pfizer reported some results from this trial – A3921133 — and the news was alarming. Unlike prior studies, this tri- al enrolled patients who were age 50 and up and had at least one cardiovascular risk factor, the company noted. The study test- ed Xeljanz on top of background metho- trexate and included an arm with patients on a tumor necrosis factor inhibitor (TNFi). Results were monitored by an external Rheumatology Drug Safety Monitoring Board (DSMB) “Based on the most recent analysis of the ongoing A3921133 study, the DSMB observed that patients treated with to- facitinib 10 mg twice daily had a statisti- cally and clinically important difference Shadows Cast Over JAK Class in the occurrence of pulmonary em- bolism, compared with patients in this Following Xeljanz Mortality Signal study who were treated with a TNFi. The DSMB also noted an increase in overall EMILY HAYES [email protected] mortality in the 10 mg twice daily treat- ment group compared to the tofacitinib he cardiovascular risks seen with a in 2016. (Also see “PIPELINE WATCH – 10 5 mg twice daily and TNFi treatment higher-than-approved dose of Pfiz- Approvals, Two Breakthroughs And A Re- arms,” Pfizer said. T er Inc.’s Janus kinase (JAK) inhibitor fusal To File Letter” - Scrip, 1 Mar, 2016.) The This safety signal has not been picked Xeljanz in a long-term rheumatoid arthri- drug’s initial clearance came with a lot of up in adverse event reporting systems, tis study required by the US FDA have cast safety baggage. A boxed warning notes according to the company. The A3921133 a shadow over the drug’s other indications risk for serious infections and labeling trial is continuing and is still blinded, but and for the class overall. advised regular monitoring of lipid levels, the design has been modified so that Xeljanz (tofacitinib) has been approved because the drug is associated with an in- participants will no longer be getting the by the FDA since 2012 at the dose of 5 mg crease in cholesterol and therefore has a higher dose. twice daily for moderate-to-severe rheuma- potential risk of cardiovascular (CV) safety “The DSMB stated it firmly believes toid arthritis (RA) after inadequate response events, notably pulmonary embolism (PE). that the risk-benefit profile of tofacitinib or intolerance to methotrexate. (Also see (Also see “Panel Backs Pfizer’s Tofacitinib 5 mg twice daily in comparison to the “Oral Dosing May Not Be Enough To Win For RA, With Narrower Indication” - Pink TNFi group remains appropriately bal- Speedy Adoption Of Pfizer’s RA Pill Xeljanz” - Sheet, 9 May, 2012.) anced in this study. We will work with Pink Sheet, 12 Nov, 2012.) The FDA did not approve a higher 10 mg the FDA and other regulatory agencies An extended release formulation of twice-daily dose and required the compa- to review the full results upon comple- tofacitinib – Xeljanz XR – was approved ny to do a new clinical study post-approval CONTINUED ON PAGE 4 BROUGHT TO YOU BY THE EDITORS OF PHARMASIA NEWS, START-UP AND SCRIP INTELLIGENCE Intercept Surges Ahead BioMarin Buyout Target Bayer Bags Vitrakvi OCA will be filed for approval Morningstar report suggests German firm takes full control of in NASH later this year (p6) company could be snapped up (p13) larotrectinib rights and revenues (p15) IN THIS ISSUE from the editor [email protected] About six years ago, the first gene therapy was approved Marin’s CEO reckons that should it win approval, the in Europe. Five years later it was withdrawn, and it was company’s hemophilia A gene therapy could still repre- never filed in the US. sent good value at $2-3m – even though the disease is Glybera, for an ultra-rare hereditary disease that caus- much more common than lipoprotein lipase deficien- es attacks of severe pancreatitis, was pulled from the cy, which Glybera treated (see p8). And Novartis has market by its maker uniQure amid uncertainty over its suggested that $4-5m would be a cost-effective price effectiveness and resistance to its price tag of around for its yet to be approved spinal muscular atrophy gene €1m. Since its launch price caused uproar in 2015, much therapy Zolgensma. progress has been made in the field of gene therapy, even While cost-effectiveness bodies – notably ICER in re- as Glybera itself crept off the stage in a puff of ignominy. cent days – continue to challenge such prices, the focus But whereas in other industries the expansion of tech- of the wider debate has shifted from outraged resist- nology leads to costs coming down, the aspect of Gly- ance to more nuanced consideration of the parameters bera that once seemed so outrageous – its price – now for measuring value and mechanisms for sharing risk seems less, not more, shocking. and spreading payment. Spark Therapeutics launched its gene therapy to And with more gene therapy makers triangulating treat a rare form of blindness at $850,000 last year. Bio- around price, tolerance to the costs looks set to increase. LEADERSHIP ADVERTISING DESIGN Phil Jarvis, Mike Ward, Christopher Keeling Paul Wilkinson Karen Coleman DESIGN SUPERVISOR SUBSCRIPTIONS Gayle Rembold Furbert Scrip Dan Simmons, Shinbo Hidenaga EDITORS IN CHIEF Andrea Charles EDITORIAL OFFICE Ian Haydock (Asia) John Davis Christchurch Court Eleanor Malone (Europe) Kevin Grogan 10-15 Newgate Street Denise Peterson (US) Ian Schofield London, EC1A 7AZ Vibha Sharma CUSTOMER SERVICES Joanne Shorthouse EXECUTIVE EDITORS US Toll-Free: +1 888 670 8900 COMMERCIAL Sten Stovall US Toll: +1 908 547 2200 Alexandra Shimmings (Europe) UK & Europe: +44 (20) 337 73737 Mary Jo Laffler (US) US Australia: +61 2 8705 6907 Michael Cipriano POLICY AND REGULATORY Japan: +81 3 6273 4260 Derrick Gingery Maureen Kenny (Europe) Email: clientservices@ Joseph Haas Nielsen Hobbs (US) pharma.informa.com Emily Hayes ASIA Mandy Jackson Cathy Kelly TO SUBSCRIBE, VISIT Anju Ghangurde scrip.pharmaintelligence.informa.com Jung Won Shin Jessica Merrill TO ADVERTISE, CONTACT Brian Yang Brenda Sandburg Bridget Silverman [email protected] Sue Sutter EUROPE All stock images in this publication Neena Brizmohun courtesy of www.shutterstock.com Francesca Bruce unless otherwise stated Scrip is published by Informa UK Limited. ©Informa UK Ltd 2019: All rights reserved. ISSN 0143 7690. 2 | Scrip | 1 March 2019 © Informa UK Ltd 2019 Gene Therapy Pricing Keytruda Liver Loss 20 4 8 Japanese iPSC Trial 11 exclusive online content inside: COVER / Shadows Cast Over JAK Class Following Xeljanz Novo Nordisk CSO: Hemophilia Treatment Mortality Signal ‘Segmenting’ Much Like In Diabetes 3 Novo Nordisk CSO: Hemophilia Treatment STEN STOVALL [email protected] ‘Segmenting’ Much Like In Diabetes 4 Merck’s Keytruda Loss In Liver Cancer Could Be Gain For Rivals 6 Intercept Retakes The Lead In NASH 8 BioMarin CEO Suggests Hemophilia Gene Therapy Pricing In $2m-$3m Range 9 Cell Therapies Forge Ahead In Japan with Reimbursement, Approval Decisions 11 Another World-First As Japan OKs Spinal Injury Cell Therapy Trial 12 Tanezumab Dances Through Back Pain Studies With “Acceptable” Safety Answers With earlier than expected FDA approval now in – and European passage expected soon for Novo Nordisk 13 BioMarin Is Buyout Target, Morningstar Says AS’s long-acting Factor VIII replacement therapy Esperoct 15 Bayer Bags Full Vitrakvi Rights As Lilly Signs (turoctocog alfa pegol), the Danish company has brought Off Loxo Buy its R&D programs based on clotting factor deficiencies to a “successful result”, says chief science officer Mads Krogs- 16 MPM Raises $400m To Build Early-Stage Biotechs gaard Thomsen. 17 Shire Solid But Hemophilia Challenges Looming? But Novo Nordisk will not be able to launch the prod- uct in the US before 2020, due to third-party intellectual 18 IQVIA Could Be CRO Bellwether As Tech Offering property agreements. In the meantime, its efforts in treat- Outperforms Expectations ing the condition will turn next to studying its humanized mAb concizumab, Thomsen told Scrip. 19 AZ China Growth Pops Again But Clouds Loom Formerly known as N8-GP, Esperoct is an extended half- 20 Sanofi Releases Annual Drug Price Report Ahead life Factor VIII for treatment of people with hemophilia Of Senate Hearing A. The molecule is a glyco-PEGylated Factor VIII in which the B-domain, a region within the factor, is deleted. It has 21 GSK’s Terrell On ‘Relentless’ Digital Consumption Across now been approved in the US for adults and children with The Spectrum hemophilia A as a routine prophylaxis to reduce the fre- 22 Pipeline Watch quency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative manage- 23 Appointments ment of bleeding. Published online 20 February 2019 To read the rest of this story go to: https://bit.ly/2U89tGf @PharmaScrip /scripintelligence /scripintelligence /scripintelligence scrip.pharmaintelligence.informa.com 1 March 2019 | Scrip | 3 HEADLINE NEWS/LIVER CANCER CONTINUED FROM PAGE 1 rent labeling “mentions four cases of lower dose, which appears to be associ- tion of this study. Furthermore, the PE observed in an UC long-term exten- ated with lower risk for thrombosis. (Also DSMB stated that other, ongoing stud- sion study.” see “AbbVie Soothes Safety Fears With More ies of tofacitinib in RA, juvenile idio- Upadacitinib RA Data” - Scrip, 7 Jun, 2018.) pathic arthritis, psoriatic arthritis, and POTENTIAL FALLOUT FOR “AbbVie’s decision to submit only the ankylosing spondylitis should continue OTHER JAK INHIBITORS low dose of upadacitinib to the FDA ap- unchanged,” the company added. In a Feb.
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