Regulatory Recon: FDA Approves Celgene's Targeted AML Drug Idhifa; Vertex Picks Up Expanded Indication for Kalydeco (1 August 2017)

Posted 01 August 2017 By Michael Mezher

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

In Focus: US

  • FDA Approves Celgene's Targeted AML Drug Idhifa (FDA) (Endpoints)
  • AstraZeneca gets breakthrough status for blood cancer drug (Reuters) (Press)
  • AstraZeneca a tricky takeover target after big cancer drug blow (Reuters)
  • Vertex wins new Kalydeco indication, ups sales forecast (BioPharmaDive) (Press)
  • Sanders to unveil rule requiring drug companies to set fair prices (The Hill) (STAT)
  • If he wanted to, Trump could take steps to lower soaring drug prices (LA Times)
  • FDA pushes to bring order to the chaotic world of DNA sequencing (STAT)
  • Indivior gets a speedy review for opioid addiction drug as FDA vows to fight a crisis (Endpoints)
  • Looking for an oncology reboot, Sanofi, Regeneron shoot for a fast and flashy FDA OK of a promising new PD-1 (Endpoints)
  • More than a third of US adults prescribed opioids in 2015 (Reuters)
  • Bristol-Myers Squibb's Opdivo nabs MSI-H colorectal cancer nod, but it can't match Keytruda's umbrella approval (Fierce) (Press)
  • Pfizer revenue misses as demand falls for older drugs, Prevnar (Reuters) (The Street) (Financial Times) (Press)
  • Express Scripts to cover Mylan's EpiPen, exclude rivals (Reuters)
  • White House Commission on Opioids to Trump: Declare a National Emergency (Focus) (NYTimes)
  • CRISPR Pioneer Zhang Preaches Extra Caution In Human Gene Editing (Xconomy) (GenomeWeb) (Nature)
  • Calls grow to tap the gold mine of human genetic knockouts (Nature)

Sponsored Content: IVDR/MDR Challenges Top Roadshow Agenda

  • BSI's 6th Annual, full-day medical device Roadshow will guide participants to new EU Medical Device/In-Vitro Device Regulation compliance. The program examines key issues facing Med Dev Manufacturers under the new MDR/IVDR starting with an overview, then analyzing legacy products and transitional provisions. Implementation issues including conformity assessments, essential/clinical requirements, SPR, labeling and QMS impacts of these standards will be detailed.
  • Check here for specific dates, locations and the program agenda.

In Focus: International

  • 19 European Cities Vie to Host EMA Headquarters (Focus) (Politico)
  • Chiesi dumps uniQure's hemophilia B gene therapy (Fierce)
  • Only Six Nations Have Evaluated Readiness for Global Pandemic (NYTimes)
  • Asia Regulatory Roundup: Australia Proposes to Align Device Regulations With EU (Focus)
  • India raises concerns around Chinese firm Fosun's takeover of Gland: Source (Economic Times)
  • Malaysia pilots barcoding to fight falsified medicines (Securing Industry)
  • Patients at risk in Brexit negotiations, warns Alliance (PharmaTimes) (Pharmafile)
  • Sanofi's eczema rollout Dupixent hits blockbuster launch trajectory as earnings beat (Fierce)
  • UK babies to be routinely immunised against hepatitis B (Pharmafile)
  • Biotechnology: the US-China dispute over genetic data (Financial Times)
  • Chinese FDA Answers Two Common Clinical Trial Questions (Emergo)

Pharmaceuticals & Biotechnology

  • Loxo beefs up its genetics-based cancer drug pipeline with an unusual $40M deal (Endpoints)
  • A fast-moving entry in the gene editing race is speeding to the clinic, fueled by an $83.5M round (Endpoints)
  • Pfizer's Goettler On What Partners Look For In Orphan Drug Deals (SCRIP-$)
  • New System Speeds FDA Import Decisions (FDAVoice)
  • Conquering HIV's capsid (C&EN) (In The Pipeline)
  • FDA Offers Guidance on Final Rule for Antiseptic Washes (Focus)
  • FDA Warns Italian Drugmaker for GMP Violations (Focus)
  • Gene therapy's 2017 scorecard – No alarms and no surprises (please) (ORF)
  • 23andMe is recruiting 25,000 people for a study on depression and bipolar disorder (CNBC)
  • HIV pre-exposure prophylaxis can be taken as needed (Reuters)
  • PhRMA CEO discusses medicine costs with CNBC's Squawk Box (PhRMA)
  • FDA Guidance on IRB Waiver or Alteration of Informed Consent for Minimal Risk Clinical Investigations: A Holdover until Rulemaking (FDA Law Blog)
  • Animal Generic Drug User Fee Rates and Payment Procedures for Fiscal Year 2018 (FDA)
  • FDA advances two more Herceptin biosims—this time from Amgen and Teva—as Roche braces for impact (Fierce)
  • Boehringer Ingelheim break ground on $217 million Fremont facility (Pharmafile)
  • New Part B Buy-and-Bill Data: Physician Offices Are Losing to Hospital Outpatient Sites (Drug Channels)
  • Unfazed by competition, Allergan sees room at CGRP table (BioPharmaDive)
  • Bicycle Therapeutics Expands Leadership Team with Appointment of Lee Kalowski as Chief Financial Officer (Press)
  • Incyte Reports 2017 Second-Quarter Financial Results and Updates on Key Clinical Programs (Press)
  • Labcorp Buying CRO Chiltern For $1.2B (BioCentury)
  • Scientists develop scorpion 'milking machine' for disease research (Reuters)
  • Pharma And Health Reform: Staying On The Sidelines Works Out (Pink Sheet-$)
  • The Drug Quality and Security Act – is the FDA walking the walk? (PharmaLetter-$)

Pharmaceuticals and Biotechnology: Study Results, Filings and Designations

  • Milestone Pharma preps a leap into PhIII cardio study with $55M round backed by Novo (Endpoints) (Fierce)
  • Kitov Submits New Drug Application to FDA for KIT-302 (Press)
  • Flex Pharma Initiates ALS Phase 2 Trial with FLX-787 in US (Press)
  • GenSight Biologics Completes Enrollment of RESCUE Phase III Study of GS010 in the Treatment of Leber's Hereditary Optic Neuropathy (Press)
  • Acceleron Announces First Patient Treated in Phase 2 Clinical Trial of ACE-083 in Charcot-Marie-Tooth Disease (Press)
  • Transgene: First Patient Dosed in a Phase 1/2 Trial of Pexa-Vec + Opdivo® for the First-Line Treatment of Advanced Liver Cancer (Press)
  • Spectrum Pharmaceuticals Announces Completion of Enrollment in the Phase 3 Pivotal Study (ADVANCE) of ROLONTIS™ (eflapegrastim), a Novel Long-Acting GCSF (Press)
  • Protalex Completes Data Analysis of Second Dose Cohort of U.S. Phase 1/2 Study of PRTX-100 in ITP (Press)
  • Sirnaomics Leading siRNA Therapeutic Candidate, STP705, Granted Orphan Designation for the Treatment of Primary Sclerosing Cholangitis (PSC) (Press)

Medical Devices

  • Bruker Receives FDA Approval for Expanded MALDI Biotyper (GenomeWeb)
  • FDA roundup: Dictum Health, Philips, Osprey Medical, VitalConnect (mobihealthnews)
  • Accuray wins FDA nod for iDMS data management system (MassDevice)
  • Boston Scientific to end Renuvia bioresorbable coronary stent program (MassDevice)
  • Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; De Novo Classification Process (Evaluation of Automatic Class III Designation) (FDA)

US: Assorted & Government

  • Senate HELP Committee Nominations Hearing for Several Health Related Posts (Senate HELP)
  • Jury to Decide: Were Shkreli's Gambles to Save His Drug Company Criminal? (NYTimes)
  • The Rule of Law is the Best Medicine, Part 2 (Drug & Device Law)
  • Berkeley Files Opening Brief in CRISPR Appeal (Patent Docs)
  • Is there a Fourth Amendment Expectation of Privacy in Prescription Records? According to the Utah District Court, Maybe Not (Harvard Bill of Health)
  • Post-BMS Personal Jurisdiction Cheat Sheet (Drug & Device Law)
  • Complaint Letter to the OHRP and the VA Office of Research Oversight Regarding an Unethical Clinical Trial Involving Heart Attack Patients (Public Citizen)
  • Bristol-Myers, Pfizer come up victorious in 20 more Eliquis bleeding cases (Fierce)
  • Insurers to fill Obamacare gaps in Ohio's individual market (Reuters)
  • Pharma, device manufacturers worry government-sponsored hackers will target research, financial information (Fierce)

Upcoming Meetings & Events

Europe

  • VNS Therapy Receives CE Mark for Expanded MRI Labeling (Press)
  • Torbay Pharmaceuticals unveils new manufacturing facility (Pharmafile)
  • First biotech neurotrophic keratitis drug Oxervate approved in Europe (PharmaLetter-$)
  • Onxeo sells legacy products to focus on orphan oncology drugs (PharmaLetter-$)
  • Myomo wins CE Mark for next-gen MyoPro powered orthotic (MassDevice)

Asia

  • Is Japan's market a black hole? (PharmaLetter-$)
  • Philippines has highest HIV infection growth rate in Asia-Pacific: U.N. (Reuters)
  • Japan clears CardioFocus' HeartLight (MassDevice)

India

  • Glenmark Pharma releases investigational skin findings (Economic Times)
  • Lupin says it fixed issue flagged by US FDA during Aurangabad audit (InPharmaTechnologist)
  • NPPA fixes prices of 67 formulations of Sch-I under DPCO 2013, retail price of 7 other formulations (PharmaBiz)
  • Applicability of WPI not correct if fixation of price of product become scheduled for first time: DoP (PharmaBiz)

Australia

  • Biosimilar Renflexis PBS listing brings potential for health budget savings for Australia (PharmaLetter-$)

General Health & Other Interesting Articles

  • A Dangerous, 'Silent Reservoir' for Gonorrhea: The Throat (NYTimes)
  • Scientists Edge Closer To Elusive Lab Test For Chronic Fatigue Syndrome (NPR) (Reuters)

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 news@raps.org.

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

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