Recon: FDA Approves Jacobus’ Ruzurgi for Children With LEMS

• FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder (FDA) (Endpoints)
• In a crafty move, FDA may have found a way to dampen controversy over a $375,000 rare-disease drug (STAT)
• Allergan lifts outlook after Botox plumps quarterly earnings (Financial Times)
• Allergan chief Saunders takes a painful $2.5B hit on an ugly PhIII flop (Endpoints)
• Alphabet's GV leads funding in gene editing company Verve Therapeutics (Reuters)
• The Birth-Tissue Profiteers (ProPublica)
• FDA hits Acacia with another rejection, cites manufacturer issues (PharmaTimes)
• Akorn lenders will declare it in default if a new loan agreement is not reached but progress is being made (Fierce)
• FDA grapples with AI medical devices (Roll Call)
• How to save $80 billion a year on prescription drugs (Axios) (STAT) (Health Affairs)
• There’s a high cost to making drugs more affordable for Americans (Washington Post)
• A $2 Million Drug Is About to Hit the Market (WSJ)
• Trump directs health chief to work on allowing Florida to import cheaper drugs (The Hill) (Politico)
• Stanley Crooke on finally making sense out of antisense (C&EN)

• GW Pharma's epilepsy drug meets main goal in late-stage trial (Reuters) (Endpoints)
• Tencent Starts Collecting Medical Data On UK Patients In Parkinson’s Trial (Forbes) (Financial Times)
• WHO issues warning as measles infects 34,000 in Europe this year (Reuters)
• Germany Considers Fines for Not Vaccinating Children Against Measles (NYTimes)
• Health Secretary Matt Hancock says he will not rule out 'compulsory vaccination' (Pharmafile)
• It’s time to stop murder by counterfeit medicine (STAT)
• Belgium’s liver-focused stem cell therapy developer Promethera raises $44M+ (Endpoints)
• EU nod for BioMarin’s second PKU therapy Palynziq (PMLive)
• EC Approves Pfizer’s Lorviqua for ALK-Positive Advances NSCLC (Press)
• Dupixent® (dupilumab) Approved for Severe Asthma by European Commission (Press)
• European Medicines Agency closed 9 May 2019 (EMA)

• High levels of sunscreen ingredients end up in the bloodstream: study (Reuters)
• 'Pathbreaking' Ionis trial offers way forward in Huntington's disease (BioPharmaDive)
• With a superstar cardiologist as its CEO, new CRISPR company targets heart disease (STAT) (Endpoints) (Xconomy)
• FDA Raises Concerns With Novartis in Clinical Inspection (Focus)
• FDA to Study DTC Ads for Drugs Approved Under Accelerated Process (Focus)
• ICER Chief Scientific Officer Bradt Brings Pharma Perspective (Pink Sheet-$)
• Testosterone Products Need Pediatric PK Studies At Minimum, US FDA Panel Says (Pink Sheet-$)
• How Do You Improve On A Cure? US FDA Panel Discusses Enrollment Conundrum (Pink Sheet-$)
• Chiesi Takes CF Product To Advisory Cmte. With Modest Efficacy, Many Questions (Pink Sheet-$)
• Insurers Brace For Pfizer's $225K Heart Drug And Whether To Pay For It (Forbes)
• Nostrum Laboratories raises price of cold remedy by 300% (Financial Times)
• Targeting cancer-associated neutrophils (Nature)
• Chronic opioid therapy needs to be individualized, but most people aren't getting that (The Hill)
• $250M AstraZeneca spinoff preps leap into 3-way battle with titans for a rare disease market that spans the globe (Endpoints)
• Bioengineers And Designers Bring 3-D-Printed Organs One Step Closer (Forbes)
• New Biktarvy spot puts Gilead in top TV-spending ranks, right behind AbbVie and Pfizer (Fierce)
• Rattle and hum: Roche, PTC post update on their SMA drug as a shaken Biogen faces yet another franchise buster (Endpoints) (Fierce) (Press)
• Watch out AbbVie and J&J, AstraZeneca just took another big swing at one of your star cancer drug franchises (Endpoints)
• Top MedImmune research exec joins the team now running Immunocore in wake of an R&D restructuring at AstraZeneca (Endpoints)
• Confo raises €30M A round to target hard-to-drug GPCRs (Fierce)
• Promethera raises €40M to fund trial of NASH cell therapy (Fierce)

• GenSight Biologics Announces Positive Data Safety Monitoring Board Review and Continuation of PIONEER Phase I/II Clinical Trial of GS030 Combining Gene Therapy and Optogenetics for the Treatment of Retinitis Pigmentosa (Press)
• Cancer Prevention Pharmaceuticals (CPP) and Mallinckrodt Announce Results from Pivotal Phase 3 Trial of CPP-1X/Sulindac in Patients with Familial Adenomatous Polyposis (Press)
• Merck’s BELSOMRA® (suvorexant) C-IV Meets Primary Efficacy Endpoint in Phase 3 Trial for the Treatment of Insomnia in People with Mild-to-Moderate Alzheimer’s Disease Dementia (Press)
• Clover Biopharmaceuticals Receives NMPA Approval for SCB-313 (TRAIL-Trimer) to Initiate Clinical Trials in China (Press)
• Sensei Biotherapeutics Announces Clinical Trial Collaboration Agreement with AstraZeneca for Two Phase 2 Studies of SNS-301 (Press)
• Ra Pharmaceuticals Announces Positive gMG Phase 2 and Open-Label, Long-Term Extension Data at the 2019 AAN Annual Meeting (Press)
• Moleculin Announces Additional Positive Interim Results in First Cohort of Phase 1/2 Clinical Studies of Annamycin in Acute Myeloid Leukemia in Europe (Press)
• Mati Therapeutics Phase II Nepafenac Evolute® Clinical Trial Results Show Significant Post-Operative Pain Reduction in Cataract Surgery Patients (Press)

• FDA Finalizes Q-Submission Feedback and Meetings Guidance (Focus)
• Prospective Data Demonstrates Effective Pain Relief and Improved Function Using Medtronic's Evolve(SM) Workflow with Spinal Cord Stimulation (Press)
• Boston Scientific Launches New Stent for Venous Obstructive Disease (MDDI)
• Class 1 Device Recall Neuro Omega (FDA)
• FDA Grants Breakthrough Device Designation to Natera's Signatera Test (Press)

• House to Vote on Bills to Update FDA’s Purple and Orange Books This Week (Focus)
• Amgen Fails To Block Cipla’s Generic Cinacalcet In US, And It May Change How Patent Settlements Are Drafted (Pink Sheet-$)
• GNC Misleads Consumers On FDA Approval, Suit Says (Law360-$)
• Apotex, Fresenius Kabi Settle Amgen's Cancer Drug IP Suit (Law360-$)
• Meth Vs. Opioids: America Has Two Drug Epidemics, But Focuses On One (KHN)
• Bennet: Medicare for All supporters 'need to level with the American people' (Politico)
• Georgia governor to sign 6-week abortion ban (Politico)
• Will the Speaker’s office finally spill the beans on its drug pricing plan? (STAT)
• You Don’t Change Horses in Mid-Stream: Product-Specific Guidances (FDA Law Blog)
• Farewell and Adieu (Drug & Device Law)
• Endo Pharmaceuticals Inc. v. Actavis LLC (Fed. Cir 2019) (Patent Docs)

• FDA Advisory Committee Calendar

• Exporting active substance manufactured in the UK in a no deal scenario (MHRA)
• UK NICE To Pharma Firms: Up Your Game In Horizon Scanning (Pink Sheet-$)
• Russia’s Biocad ready to commission new flagship facility in St Petersburg region (PharmaLetter-$)

• Asia Regulatory Roundup: China Reviews Supervision of Cell and Gene Therapies Through Regulatory Action Plan (Focus)

• Canadian regulators issue recommendations for 3D-printed Medical Device License applicants (Emergo)
• Keytruda - Notice of Compliance with Conditions - Qualifying Notice (Health Canada)