CY 2020 CDER Drug and Biologic Calendar Year Priority Approvals As of December 31, 2020
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Ripretinib Demonstrated Activity Across All KIT/PDGFRA Mutations In
Ripretinib demonstrated activity across all KIT/PDGFRA mutations in patients with fourth-line advanced gastrointestinal stromal tumor: Analysis from the phase 3 INVICTUS study Patrick Schöffski1, Sebastian Bauer2, Michael Heinrich3, Suzanne George4, John Zalcberg5, Hans Gelderblom6, Cesar Serrano Garcia7, Robin L Jones8, Steven Attia9, Gina D’Amato10, Ping Chi11, Peter Reichardt12, Julie Meade13, Kelvin Shi13, Ying Su13, Rodrigo Ruiz-Soto13, Margaret von Mehren14, Jean-Yves Blay15 1University Hospitals Leuven, Leuven, Belgium; 2West German Cancer Center, Essen, Germany; 3OHSU Knight Cancer Institute, Portland, OR, USA; 4Dana-Farber Cancer Institute, Boston, MA, USA; 5Monash University, Melbourne, VIC, Australia; 6Leiden University Medical Center, Leiden, Netherlands; 7Vall d’Hebron Institute of Oncology, Barcelona, Spain; 8Royal Marsden and Institute of Cancer Research, London, UK; 9Mayo Clinic, Jacksonville, FL, USA; 10Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA; 11Memorial Sloan Kettering Cancer Center, New York, NY, USA; 12Sarcoma Center, Helios Klinikum Berlin-Buch, Berlin, Germany; 13Deciphera Pharmaceuticals, LLC, Waltham, MA, USA; 14Fox Chase Cancer Center, Philadelphia, PA, USA; 15Centre Leon Berard, Lyon, France KIT mutation analysis by combined tumor and liquid biopsy INTRODUCTION RESULTS Figure 7. Hazard ratio of PFS with different mutation groups by combined • Patients were grouped into 4 subsets: any KIT exon 9, any KIT exon 11, any KIT exon 13, and any KIT exon 17 tumor and liquid biopsy • Patients -
Cstone's Partner Blueprint Medicines Presented Updated Part 1 Data from PIONEER Trial of Avapritinib
Hong Kong Exchanges and Clearing Limited and The Stock Exchange of Hong Kong Limited take no responsibility for the contents of this announcement, make no representation as to its accuracy or completeness and expressly disclaim any liability whatsoever for any loss howsoever arising from or in reliance upon the whole or any part of the contents of this announcement. The forward-looking statements made in this announcement relate only to the events or information as of the date on which the statements are made in this announcement. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this announcement completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this announcement, statements of, or references to, our intentions or those of any of our directors and/or our Company are made as of the date of this announcement. Any of these intentions may alter in light of future development. CStone Pharmaceuticals 基 石 藥 業 (Incorporated in the Cayman Islands with limited liability) (Stock Code: 2616) VOLUNTARY ANNOUNCEMENT CSTONE’S PARTNER BLUEPRINT MEDICINES PRESENTED UPDATED PART 1 DATA FROM PIONEER TRIAL OF AVAPRITINIB SHOWING ROBUST REDUCTIONS IN CUTANEOUS DISEASE SYMPTOMS IN PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS The partner of CStone Pharmaceuticals (the “Company” or “CStone”), Blueprint Medicines Corporation (NASDAQ: BPMC) (“Blueprint Medicines”), announced on June 6 updated clinical data from part 1 of the PIONEER trial showing robust and consistent clinical activity for avapritinib across multiple qualitative and quantitative measures of cutaneous disease in patients with indolent systemic mastocytosis (“ISM”). -
Clinical Policy: Sorafenib (Nexavar)
Clinical Policy: Sorafenib (Nexavar) Reference Number: CP.PHAR.69 Effective Date: 07.01.11 Last Review Date: 05.21 Line of Business: Commercial, HIM, Medicaid Revision Log See Important Reminder at the end of this policy for important regulatory and legal information. Description Sorafenib (Nexavar®) is a kinase inhibitor. FDA Approved Indication(s) Nexavar (sorafenib) is indicated for the treatment of: Unresectable hepatocellular carcinoma (HCC); Advanced renal cell carcinoma (RCC); Locally recurrent or metastatic, progressive, differentiated thyroid carcinoma (DTC) that is refractory to radioactive iodine treatment. Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria. It is the policy of health plans affiliated with Centene Corporation® that Nexavar is medically necessary when the following criteria are met: I. Initial Approval Criteria A. Hepatocellular Carcinoma (must meet all): 1. Diagnosis of HCC; 2. Prescribed by or in consultation with an oncologist; 3. Age ≥ 18 years; 4. Confirmation of Child-Pugh class A or B7 status; 5. Request meets one of the following (a or b):* a. Dose does not exceed 800 mg per day; b. Dose is supported by practice guidelines or peer-reviewed literature for the relevant off-label use (prescriber must submit supporting evidence). *Prescribed regimen must be FDA-approved or recommended by NCCN Approval duration: Medicaid/HIM – 6 months Commercial – Length of Benefit B. Renal Cell Carcinoma (must meet all): 1. Diagnosis of advanced RCC; 2. Prescribed by or in consultation with an oncologist; 3. Age ≥ 18 years; 4. Request meets one of the following (a or b):* Page 1 of 8 CLINICAL POLICY Sorafenib a. -
Cogent Biosciences Announces Creation of Cogent Research Team
Cogent Biosciences Announces Creation of Cogent Research Team April 6, 2021 Names industry veteran John Robinson, PhD as Chief Scientific Officer New Boulder-based team with exceptional track record of drug discovery and development focused on creating novel small molecule therapies for rare, genetically driven diseases Strong year-end cash position of $242.2 million supports company goals into 2024, including three CGT9486 clinical trials on-track to start this year, beginning with ASM in 1H21 BOULDER, Colo. and CAMBRIDGE, Mass., April 6, 2021 /PRNewswire/ -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced the formation of the Cogent Research Team led by newly appointed Chief Scientific Officer, John Robinson, PhD. "Today marks an important step forward for Cogent Biosciences as we announce the formation of the Cogent Research Team with a focus on discovering and developing new small molecule therapies for patients fighting rare, genetically driven diseases," said Andrew Robbins, President and Chief Executive Officer of Cogent Biosciences. "I am thrilled to welcome John onboard as Cogent Biosciences' Chief Scientific Officer. John's expertise and seasoned leadership make him ideally suited to lead this new team of world class scientists. Given the team's impressive experience and accomplishments, we are excited for Cogent Biosciences' future and the opportunity to expand our pipeline and deliver novel precision therapies for patients." With an exceptional track record of innovation, the Cogent Research Team will focus on pioneering best-in-class, small molecule therapeutics to both improve upon existing drugs with clear limitations, as well as create new breakthroughs for diseases where others have been unable to find solutions. -
Sample of Current Attendees
SAMPLE OF CURRENT ATTENDEES TITLE COMPANY Sr Mgr Global Meetings Mgmt Amgen Prin Tradeshow Spec Boston Scientific Events Mgr PR Medical Events Sr Buyer Pharma Ops ITA Group Travel Services Supv Grifols Asia Pacific Pte Ltd Compliance Specialist GCO Spend Jouney Lead Global Mtgs Travel Events UCB Category Project Specialist Roche Events & Travel Mgr Teva Brasil Mgr Event Mgmt Bayer Congress Mgr MediCongress Services Global Events Mgmt Sr Assoc Amgen Assoc Dir Congresses & Events MSD Internal Event Mgr Gilead Sourcing Mgr BMS Dir Sourcing Meeting Alliance Custoerm Srvs Meetings Mgr Eli Lilly & Company Sr Congress Mgr Novartis Pharmaceuticals Sourcing Mgr BCD Meetings & Events Sr Buyer Meetings & Incentives Worldwide Sourcing Mgr American Express GBT Acct Mgr Venue Sourcing Ashfield Meetings & Events Mtg Planner AbbVie Sr Sourcing Mgr CWT Dir Sourcing Planning Partners International Meetings Mgr ACOG Meeting Mgr Pfizer Canada Global Event Lead Astellas Meeting & Events Sanofi Events Specialist Cook Medical Meetings & Events Mgr Pfizer Spain Pfizer Spain Sr Mgr Hotel Procurement Meetings & Incentives WorldWide Inc Event Planner SFU BCcampus Mgr Global Strategic Mtg Mgmt Teva Pharmaceuticals Global Mtgs & Events Virtual Mtgs Specialist AstraZeneca Global Congress Strategy Lead MSD Assoc Dir Congresses & Events Alnylam Pharmaceuticals Event Planner AbbVie Institutional & Scientific Events Coordr Almirall Congress Mgr AO Spine Sr Meeting Planner Medtronic Mgr Global Mtgs Mgmt Alnylam Pharmaceuticals Meeting Planner AbbVie Global Audit Meetings -
View State-Of-The-Art Clinical Symposium Disclosures
Planning Committee Members The following Planning Committee members have no relevant financial relationship(s) with ineligible companies to disclose. Mastering E/M Changes for 2021 • Antanya Chung-Gardiner, BSc • Melesia Tillman 2021 Access in Rheumatology Planning Committee • Marcus Snow, MD 2021 Fellow-in-Training Educational Session Planning Committee • Nadia Elias, MD • Tate Johnson, MD • Justin Levinson, MD, MBA • Jean Lin, MD, PhD • Megan Lockwood, MD • Tyler Reese, MD • Zahra Rehman, MD • Didem Saygin, MD • Rachel Wallwork, MD 2021 State-of-the-Art Clinical Symposium Planning Committee • Sobia Hassan, MD • Aman Kugasia, MD • Jeanie Lee, MD • Manjari Malkani, MD • Rebecca Manno, MD, MHS The following Planning Committee members have relevant financial relationship(s) with ineligible companies to disclose. 2021 Access in Rheumatology Planning Committee • Christopher Phillips, MD - Pfizer (Self): 5 2021 Fellow-in-Training Educational Session Planning Committee • Mary Mamut, DO - Bendcare (Self): 8 2021 State-of-the-Art Clinical Symposium Planning Committee • Rohit Aggarwal, MD, MS - AbbVie, Amgen, , Genzyme, , Novartis, Roche, Sandoz, UCB (Self): 1, 5, 2; AdMIRx, Inc. / Now Q-32 (Self): 5, 2; Alexion (Self): 5; Argenx (Self): 5; AstraZeneca (Self): 5; Boehringer Ingelheim (BI) (Self): 5; Bristol-Myers Squibb(BMS) (Self): 5, 2; Corbus (Self): 5; CSL Behring (Self): 1, 5; EMD Serono (Self): 5, 2; Genentech (Self): 2; Janssen (Self): 5; Kezar (Self): 5; Kyverna (Self): 5; Mallinckrodt (Self): 1, 2; Octapharma (Self): 1, 5; Orphazyme (Self): 1; Pfizer (Self): 5, 2; Scipher Medicine: 5 • Narender Annapureddy, MD, MS - Medpage (Self): 1, Expert Reviewers for the ACR Reading Room section of MedPage Today. • Lianne Gensler, MD - AbbVie (Self): 1, 5; Eli Lilly (Self): 5; Gilead: 5; GSK (Self): 5; Janssen (Self): 5; Novartis (Self): 5; Pfizer (Self): 5, 2; UCB: 5 Faculty The following Faculty members have no relevant financial relationship(s) with ineligible companies to disclose. -
First-Line Treatment Options for Patients with Stage IV Non-Small Cell Lung Cancer with Driver Alterations
First-Line Treatment Options for Patients with Stage IV Non-Small Cell Lung Cancer with Driver Alterations Patients with stage IV non-small cell lung cancer Nonsquamous cell carcinoma and squamous cell carcinoma Activating EGFR mutation other Sensitizing (L858R/exon 19 MET exon 14 skipping than exon 20 insertion mutations, EGFR exon 20 mutation ALK rearrangement ROS1 rearrangement BRAF V600E mutation RET rearrangement NTRK rearrangement mutations KRAS alterations HER2 alterations NRG1 alterations deletion) EGFR mutation T790M, L858R or Ex19Del PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options PS 0-2 Treatment Options Emerging target; no Emerging target; no Emerging target; no Platinum doublet † † † Osimertinib monotherapy S Afatinib monotherapy M M Alectinib S Entrectinib M Dabrafenib/trametinib M Capmatinib M Selpercatinib M Entrectinib M conclusions available conclusions available conclusions available chemotherapy ± bevacizumab Gefitinib with doublet Standard treatment based on Standard treatment based on Standard treatment based on M M M Brigatinib S Crizotinib M M Tepotinib M Pralsetinib* W Larotrectinib M chemotherapy non-driver mutation guideline non-driver mutation guideline non-driver mutation guideline If alectinib or brigatinib are not available If entrectinib or crizotinib are not available Standard treatment based on Standard treatment based on Standard treatment based on Dacomitinib monotherapy M Osimertinib W M M M Ceritinib S Ceritinib W non-driver mutation guideline non-driver mutation guideline non-driver mutation guideline Monotherapy with afatinib M Crizotinib S Lortlatinib W Standard treatment based on Erlotinib/ramucirumab M M non-driver mutation guideline Erlotinib/bevacizumab M Monotherapy with erlotinib M Strength of Recommendation Monotherapy with gefitinib M S Strong M Moderate W Weak Monotherapy with icotinib M Notes. -
CDER Breakthrough Therapy Designation Approvals Data As of December 31, 2020 Total of 190 Approvals
CDER Breakthrough Therapy Designation Approvals Data as of December 31, 2020 Total of 190 Approvals Submission Application Type and Proprietary Approval Use Number Number Name Established Name Applicant Date Treatment of patients with previously BLA 125486 ORIGINAL-1 GAZYVA OBINUTUZUMAB GENENTECH INC 01-Nov-2013 untreated chronic lymphocytic leukemia in combination with chlorambucil Treatment of patients with mantle cell NDA 205552 ORIGINAL-1 IMBRUVICA IBRUTINIB PHARMACYCLICS LLC 13-Nov-2013 lymphoma (MCL) Treatment of chronic hepatitis C NDA 204671 ORIGINAL-1 SOVALDI SOFOSBUVIR GILEAD SCIENCES INC 06-Dec-2013 infection Treatment of cystic fibrosis patients age VERTEX PHARMACEUTICALS NDA 203188 SUPPLEMENT-4 KALYDECO IVACAFTOR 21-Feb-2014 6 years and older who have mutations INC in the CFTR gene Treatment of previously untreated NOVARTIS patients with chronic lymphocytic BLA 125326 SUPPLEMENT-60 ARZERRA OFATUMUMAB PHARMACEUTICALS 17-Apr-2014 leukemia (CLL) for whom fludarabine- CORPORATION based therapy is considered inappropriate Treatment of patients with anaplastic NOVARTIS lymphoma kinase (ALK)-positive NDA 205755 ORIGINAL-1 ZYKADIA CERITINIB 29-Apr-2014 PHARMACEUTICALS CORP metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib Treatment of relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients NDA 206545 ORIGINAL-1 ZYDELIG IDELALISIB GILEAD SCIENCES INC 23-Jul-2014 for whom rituximab alone would be considered appropriate therapy due to other co-morbidities -
Pushing the Limits: Being Innovative in a Regulated Industry
ISPE Boston Area Chapter Presents: Pushing the Limits: Being Innovative in a Regulated Industry Thursday, November 15, 2018 5:30 pm to 8:30 pm Alnylam 300 Third Street THANK YOU TO OUR PROGRAM SPONSOR Cambridge, MA 02142 EVENT INFORMATION: Join the ISPE Boston Area Chapter for an open forum on innovation. Push your own limits by participating in our pre-program activity with pharma colleagues while enjoying refreshments and a cash bar. Please have valid I. D. ready for a security checkpoint. Walk-ins are still welcome to register onsite. PROGRAM SUMMARY: Innovation comes in many forms ranging from scientific breakthroughs to new technologies and processes. This panel discussion will focus on innovation, and how it impacts the phases of the drug development lifecycle through regulatory compliance. The intent of this discussion is to allow for an interactive look into how innovation can be applied in a regulated field by providing an open forum for the moderator and audience to interact with industry leaders. If you want to better understand how to integrate innovation in your business, or the effects of innovation on your business, this panel will provide much-needed insight. By having people walking the walk of innovation from Development, Engineering, Manufacturing and Regulatory Affairs, we will be able to look to interdependencies, conflict, and synergies of innovative products. Topics will range from technical, personnel, and regulatory decisions needed to incorporate innovation into your business. WHO SHOULD ATTEND: Development, Engineering, Manufacturing, Quality Assurance and Regulatory Affairs personnel. Anybody that wants to better understand how to develop and execute teams when implementing innovative platforms or technologies. -
2020 Master Class Course Best Practices and Expanding Treatment Options in Soft Tissue Sarcomas Including GIST George D
2020 Master Class Course Best Practices and Expanding Treatment Options in Soft Tissue Sarcomas including GIST George D. Demetri, MD FACP FASCO 1 George D. Demetri, MD Faculty Disclosure • Scientific consultant with sponsored research to Dana-Farber: Bayer, Pfizer, Novartis, Epizyme, Roche/Genentech, Epizyme, LOXO Oncology, AbbVie, GlaxoSmithKline, Janssen, PharmaMar, Daiichi-Sankyo, AdaptImmune • Scientific consultant: GlaxoSmithKline, EMD-Serono, Sanofi, ICON plc, MEDSCAPE, Mirati, WCG/Arsenal Capital, Polaris, MJ Hennessey/OncLive, C4 Therapeutics, Synlogic, McCann Health • Consultant/SAB member with minor equity holding: G1 Therapeutics, Caris Life Sciences, Erasca Pharmaceuticals, RELAY Therapeutics, Bessor Pharmaceuticals, Champions Biotechnology, Caprion/HistoGeneX • Board of Directors member and Scientific Advisory Board Consultant with minor equity holding: Blueprint Medicines, Translate BIO • Patents/Royalties: Novartis royalty to Dana-Farber for use patent of imatinib in GIST • Non-Financial Interests: AACR Science Policy and Government Affairs Committee Chair, Alexandria Real Estate Equities, Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off label or investigational uses (any uses not approved by the FDA) of products or devices. 2 Soft Tissue Sarcomas: Approximate Anatomic Distribution Head and Neck 10% Upper extremities 15% Trunk 10% Retroperitoneal, pelvic and visceral Lower extremities 15% 50% (includes GIST and GYN sarcomas) Modified from Clark MA et al. NEJM 2005;353:701-11 3 Sarcomas represent an exceedingly diverse set of diseases Bone Sarcomas 10% GIST Un- differentiated Liposarcomas Leiomyo- Sarcomas 4 Ducimetiere et al. -
Human Sequencing Resource with UK Biobank
January 8, 2018 Regeneron Forms Consortium of Leading Life Sciences Companies to Accelerate Largest Widely-Available 'Big Data' Human Sequencing Resource with UK Biobank By end of 2019, Regeneron plans to sequence the exomes of all 500,000 people within the UK Biobank resource, all with associated health records, creating an unprecedented resource linking human genetic variations to human biology and disease AbbVie, Alnylam Pharmaceuticals, AstraZeneca, Biogen and Pfizer will join Regeneron to co-fund one of the industry's most ambitious 'pre-competitive' research efforts Exome sequencing data and findings will be openly available to other researchers TARRYTOWN, N.Y., Jan. 8, 2018 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), along with new collaborators AbbVie, Alnylam Pharmaceuticals, AstraZeneca, Biogen and Pfizer Inc., today announced the formation of a major 'pre-competitive' consortium to fund the generation of genetic exome sequence data from the 500,000 volunteer participants who make up the UK Biobank health resource. The newly announced collaborators will each commit $10 million to enable a dramatic acceleration of sequencing timelines, and additional companies are considering joining the consortium. Regeneron will conduct the sequencing effort. The sequencing data will be paired with detailed, de-identified medical and health records within the UK Biobank resource, including enhanced measures such as brain, heart and body imaging, to create an unparalleled resource for linking human genetic variations to human biology and disease. It was originally planned that sequencing of all 500,000 samples in the UK Biobank would be completed by 2022, with the first 50,000 people sequenced during 2017 with funding from Regeneron and GlaxoSmithKline. -
Overcoming MET-Dependent Resistance to Selective RET Inhibition in Patients with RET Fusion–Positive Lung Cancer by Combining Selpercatinib with Crizotinib a C Ezra Y
Published OnlineFirst October 20, 2020; DOI: 10.1158/1078-0432.CCR-20-2278 CLINICAL CANCER RESEARCH | RESEARCH BRIEFS: CLINICAL TRIAL BRIEF REPORT Overcoming MET-Dependent Resistance to Selective RET Inhibition in Patients with RET Fusion–Positive Lung Cancer by Combining Selpercatinib with Crizotinib A C Ezra Y. Rosen1, Melissa L. Johnson2, Sarah E. Clifford3, Romel Somwar4, Jennifer F. Kherani5, Jieun Son3, Arrien A. Bertram3, Monika A. Davare6, Eric Gladstone4, Elena V. Ivanova7, Dahlia N. Henry5, Elaine M. Kelley3, Mika Lin3, Marina S.D. Milan3, Binoj C. Nair5, Elizabeth A. Olek5, Jenna E. Scanlon3, Morana Vojnic4, Kevin Ebata5, Jaclyn F. Hechtman4, Bob T. Li1,8, Lynette M. Sholl9, Barry S. Taylor10, Marc Ladanyi4, Pasi A. Janne€ 3, S. Michael Rothenberg5, Alexander Drilon1,8, and Geoffrey R. Oxnard3 ABSTRACT ◥ Purpose: The RET proto-oncogene encodes a receptor tyrosine Results: MET amplification was identified in posttreatment kinase that is activated by gene fusion in 1%–2% of non–small biopsies in 4 patients with RET fusion–positive NSCLC treated with cell lung cancers (NSCLC) and rarely in other cancer types. selpercatinib. In at least one case, MET amplification was clearly Selpercatinib is a highly selective RET kinase inhibitor that has evident prior to therapy with selpercatinib. We demonstrate that recently been approved by the FDA in lung and thyroid cancers increased MET expression in RET fusion–positive tumor cells causes with activating RET gene fusions and mutations. Molecular resistance to selpercatinib, and this can be overcome by combining mechanisms of acquired resistance to selpercatinib are poorly selpercatinib with crizotinib. Using SPPs, selpercatinib with crizo- understood.