Regulatory Focus™ > News Articles > Regulatory Recon: FDA Approves First ALS Drug in Two Decades; Kite Discloses Patient Death in CAR-T

Regulatory Recon: FDA Approves First ALS Drug in Two Decades Kite Discloses Patient Death in CAR-T Study (8 May 2017)

Posted 08 May 2017 | By Michael Mezher 

Regulatory Recon: FDA Approves First ALS Drug in Two Decades Kite Discloses Patient Death in CAR-T Study (8 May 2017)

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.

In Focus: US

  • The First ALS Drug In 22 Years Is Approved -- And It Costs 4 Times What It Does In Japan (Forbes) (Reuters) (NYTimes) (FDA) (Press)
  • Drug Lobby Said to Mull Membership Cuts Amid Price Scrutiny (Bloomberg)
  • 5 questions as Trump prepares to welcome biotech execs to the White House (STAT) (Fierce)
  • Cardiac Patients in Trials Don't Reflect Real-World Populations (Medpage)
  • HHS Task Force: FDA Cyber Oversight Limited, Device Makers Treat Security As 'Afterthought' (IHP)
  • Kite Pharma Discloses CAR-T Patient Death, Rattling Investors (The Street)
  • A Medicare Drug Incentive That Leads to Greater Hospitalizations (NYTimes)
  • Health Act Repeal Could Threaten U.S. Job Engine (NYTimes)
  • A Republican Principle Is Shed in the Fight on Health Care (NYTimes)
  • FDA Official Highlights Foreign Supply Chain Challenges (Focus)
  • FDA Proposal Explains Uses for New Innovation Funds (Focus)
  • Pricing row shines spotlight on US pharmacy benefit managers (Financial Times)
  • What's likely to happen to the health bill in the Senate (Politico)
  • Struggling Durect teams up with Novartis' Sandoz in $293M pain drug deal (Fierce)
  • With $145M Buyout, River Vision's Plan For Stalled Roche Drug Pays Off (Xconomy) (Fierce)
  • Regeneron pens Inovio I-O combo cocktail pact for aggressive brain tumors (Fierce)
  • Compliance Concerns Slow Industry Uptake Of Cancer Patient-Reported Outcomes Tool (Pink Sheet-$)
  • Will Patient-Reported Data Require New Drug Labeling Framework? (Pink Sheet-$)
  • US Reliance On EU Drug Facility Inspections Begins In November (Pink Sheet-$)
  • FDA Leaders Dish On Agency Policies Under Trump (Law360-$)
  • FDA rejects TherapeuticsMD's vaginal pain drug; shares slump (Reuters) (The Street)

In Focus: International

  • GSK 'real world' drug test has second success in asthma (Reuters) (BioCentury) (PharmaTimes)
  • FT Health: Drug pricing must be reformed (Financial Times)
  • Final EU MDR, IVDR Texts Published, Countdown to Implementation Begins (Focus)
  • GHTC Calls for New Global Body to Guide Health R&D (GHTC)
  • Top 15 global pharma cos under pressure, net income drop by 16.5% in 2016 (PharmaBiz)
  • New ICH Guideline Could Hold The Key To Better Global Management Of Product Variations (Pink Sheet-$)
  • Antibody-powered nucleic acid release using a DNA-based nanomachine (Nature)
  • Implementing a two-invoice system in China's MedTech market (McKinsey)
  • Yemen cholera outbreak kills 25 people in a week: WHO (Reuters)
  • Meet the world's most powerful doctor: Bill Gates (Politico)
  • European Medicines Agency closed 9 May 2017 (EMA)
  • Turkey Cancer Drug Proposal Swims Against Cost Control Tide (Pink Sheet-$)
  • New Zealand pharma industry calls for faster reimbursement decisions (PharmaLetter-$)
  • India to restart inspections of API mfg facilities in China with huge rise in imports (PharmaBiz)
  • AMR: Fears over farm links to pharma (Financial Times)
  • Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 2-5 May 2017 (EMA)

US: Pharmaceuticals & Biotechnology

  • April Sees 66 ANDA Approvals and 112 CRLs (FDA News-$) (Lachman Consultants)
  • Soon-Shiong's top executive steps down at NantHealth (STAT)
  • Patient reported outcomes: Putting the patient first (Pharmafile)
  • FDA's Ruling on Generic ZITHROMAX 180-Day Exclusivity: A Little Something for Everyone! (FDA Law Blog)
  • Beyond Right To Try (BioCentury)
  • FDA Form 483 for Dr Reddy's Facility (FDA)
  • NYU Gets Another Shot at Earning Royalties From Pfizer's Xalkori (Bloomberg)
  • PTC Therapeutics' DMD drug Emflaza to cost $35,000 a year and launch within the coming weeks (MarketWatch)
  • Chemists May Have Found A Way To Make Pharmaceuticals Cheaper And Safer (Forbes)
  • The Weird and Wild Gross-to-Net Adventures of EpiPen and Its Alternatives (rerun) (Drug Channels)
  • FDA Tea Leaves: Are They About To Delay The November Deadline? (RxTrace)
  • Roche launches AVENIO ctDNA Analysis Kits for Oncology Research (Press)
  • FDA denies issuing order to turn TVs to Fox (CBS) (Medpage)
  • Juno hopes to make T Cells in two days using reagents and automation tech (BioPharmaReporter)
  • Third Cell's The Charm (BioCentury)

US: Pharmaceuticals and Biotechnology: Clinical Study Results, Filings & Designations

  • Ocular Therapeutix Presents Additional Phase 3 Data and Patient Reported Outcomes Results for DEXTENZA at the American Society of Cataract and Refractive Surgery (ASCRS) Symposium (Press)
  • Takeda Announces Publication of ALUNBRIG (brigatinib) Pivotal Phase 2 ALTA Clinical Trial Data in Journal of Clinical Oncology (Press)
  • Oncolytics Biotech Inc. Announces FDA Fast Track Designation for REOLYSIN in Metastatic Breast Cancer (Press)
  • FDA Grants Fast Track Designation to XyloCor Therapeutics Lead Candidate XC001 (Press)
  • Teva's Laquinimod Fails Phase III RRMS Study (BioCentury) (Fierce) (Pharmafile)
  • Pipeline Watch: Top-Line Phase III Brexpiprazole, Ferumoxytol Results (SCRIP-$)

US: Medical Devices

  • Patients with Parkinson's Get Their Say on Device Trial Design (MDDI)
  • FDA approves new Cardiovascular Systems saline pump after recall (MassDevice)
  • Analysts: Trumpcare would have 'minimal' negative effect on medtech (MassDevice)
  • LENSAR Receives FDA Clearances for Laser Cataract Platform Integration With OCULUS Pentacam Tomographers (Press)
  • Medrobotics Corporation Receives FDA Clearance for Flex Robotic System for Scarfree Colorectal Procedures (Press)
  • Crospon wins FDA 510(k) for next-gen Endoflip system (MassDevice)
  • Bovie Medical Receives 510K Clearance for its J-Plasma Precise Flex Handpiece (Press)
  • CytoSorbents touts Refresh trial study data (MassDevice)

US: Assorted & Government

  • Trump signs spending bill, averting government shutdown (Reuters)
  • "Pre-Approval" Design Defects − No Such Thing (Drug & Device Law)
  • Senator Proposes Bill to Increase Transparency of User Fees (Congress)
  • Republican health bill stirs fear in opioid-wracked West Virginia (Reuters)
  • 'Gray death' is the latest opioid street mix causing worry (AP)
  • White House Proposes Cutting Drug Control Office Funding by 95% (NYTimes)
  • Republicans Disregarded the C.B.O., but It Won't Be Ignored (NYTimes) (NPR)
  • Democrats criticize Senate's all-male healthcare group (Reuters)
  • EPA dismisses half of its scientific advisers on key board, citing 'clean break' with Obama administration (Washington Post)
  • Zimmer Biomet wins Trilogy hip implant suit (MassDevice)

Upcoming Meetings & Events


  • New decree on fees chargeable by the Finnish Medicines Agency entered into force on 1 April 2017 (FIMEA)
  • ABPI Welcomes Amended Legislation On Curbing Excessive Unbranded Drug Prices (Pink Sheet-$)
  • Sanofi decides to keep Cepia biz (Drug Delivery)
  • Lib Dems pledge to raise tax for NHS (PharmaTimes)
  • NHS staff feeling the pay squeeze, reports NHS Providers (Pharmafile)


  • Histogenics Announces Agreement With the Japan Pharmaceuticals and Medical Devices Agency Regarding Japanese Regulatory Pathway for NeoCart (Nasdaq)
  • Ping An Launches $1B Fund, Hires CEO (BioCentury)


  • Natco Pharma launches generic hepatitis C drug in India (Economic Times)
  • Call for branded drugs to be phased out in India (PharmaLetter-$)
  • IPA urges govt to adopt price monitoring of drugs instead of price control (PharmaBiz)
  • CDSCO, Maha FDA joint inspections to fix lacunae at blood banks over inadequate staff and equipment (PharmaBiz)

General Health & Other Interesting Articles

  • Mind Reading Algorithms Reconstruct What You're Seeing Using Brain Scan Data (MIT Technology Review)
  • POW: Here Comes A Tick Disease That's Worse Than Lyme (Forbes)

Regulatory Reconnaissance is our daily intelligence briefing for the regulatory affairs space, bringing you the top regulatory news stories from around the globe. Each weekday morning, we aim to bring you the latest highlights of new approvals, meetings, legal and political developments, regulations and guidance, and the latest trends with the potential to impact regulatory affairs professionals and the industry in which they work.

Need to contact the editor of Regulatory Reconnaissance? Find him on Twitter at @Michael_Mezher or send him an email at

A story's inclusion in Regulatory Reconnaissance does not imply endorsement by Regulatory Focus or RAPS.

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