Recon: FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation challenges

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

In Focus: US

• How Trump loyalist Heidi Overton became the president’s pick to run the FDA (STAT)
• Trump's pick to lead the FDA faces uphill Senate battle (Politico)
• Senate Health Panel Chairman Voices Strong Reservations About FDA Nominee (NYTimes)
• Ultragenyx's gene therapy gets FDA OK for glycogen storage disorder (Endpoints) (FDA)
• Regeneron drug for disease that causes dangerous bone growth earns FDA approval (STAT) (Regeneron)
• Vitestro secures FDA nod for robotic blood draw device (MedTech Dive) (FDA)
• FDA weighs in on the development and use of digitally derived measures for clinical investigations (FDA)
• After two decades under PMA, oncology ISH companion tests get a lighter regulatory path (Medtech Insight)
• FDA’s hands-on repurposing efforts show why a formal pathway is needed (Pink Sheet)
• FDA hands warning letters to manufacturers in China, India (Fierce Pharma)

In Focus: International

• Bristol Myers' China partner says bispecific ADC clears fourth cancer trial, this time in NSCLC (Endpoints)
• Industry could recoup fees if MHRA misses deadlines under new proposal (Pink Sheet)
• Failed inspection delays EU timeline for SMA drug apitegromab as FDA decision nears (Pink Sheet)
• MHRA to microbiome drug developers: Engage early and bring your products forward (Pink Sheet)
• UK hopes quantum breast cancer imaging can lower NHS wait times (Medtech Insight)
• Contractor SK Pharmteco heralds completion of two API plants in Ireland (Fierce Pharma)
• Why Tenet’s $30m round is really a bet on India’s diagnostics roll-up (Medtech Insight)
• CSL shares up 17% as immunoglobulin boost points to signs of recovery (Fierce Pharma)
• Congo set to receive 70,000 Ervebo vaccines to contain Ebola outbreak, WHO says (Reuters)

Pharma & Biotech

• Merck, Moderna declare Phase 3 success for personalized mRNA cancer vaccine (Endpoints) (New York Times)
• Amylin design debate heats up as Lilly's crucial mid-stage weight loss data near (Endpoints)
• Celon Pharma's generic semaglutide drug matches Ozempic in early study, shares jump (Reuters)
• New oncology clinical outcome assessment tool reinforces complexity of FDA qualification process (Pink Sheet)
• Novo kicks off trial testing smaller doses of Wegovy pill (Fierce Pharma)
• BioMarin does another small deal to get early-stage bone disease asset (Endpoints)
• Capricor Therapeutics’ Duchenne treatment is heading for an FDA rejection (STAT)
• Novonesis expects results from weight-loss drug supplement study in 2027 (Reuters)
• Boehringer Ingelheim again rises up rare disease reputation rankings (Fierce Pharma)
• SK strikes settlement with MSN over future Xcopri generic: reports (Fierce Pharma)
• WuXi Bio to offload chromatography unit; Cabaletta names another CDMO partner (Endpoints)

Medtech

• GE HealthCare names new CFO (MedTech Dive)
• FDA proposes to reclassify blue-light cystoscopy systems, opens Karl Storz–Photocure bladder cancer (Medtech Insight)
• EU to procure €2.5m of ebola tests as medtech response builds momentum (Medtech Insight)
• Try first, evaluate later: How the FDA is approaching digital health devices (Endpoints)

Food & Nutrition

• FDA upgrades recall of frozen blueberries sold at Publix to highest health risk level (CBS News)
• Cyclosporiasis cases now total 15,716: CDC (The Hill)

Government & Legal

• Federal appeals court revives part of Teva's IRA case as Austedo price cuts loom (Endpoints)
• Former Fauci aide pleads guilty to dodging federal public records laws (Politico)
• New CDC director acknowledges agency problems in first large staff meeting (AP News)
• GLP-1s may spur next wave in youth harm claims against social media (Bloomberg)

Regulatory Recon is our regular intelligence briefing for the regulatory affairs space, bringing you the top regulatory, biopharma and medtech news stories from around the globe. Regulatory Recon is published each week on Monday, Tuesday and Thursday.

A story’s inclusion in Regulatory Recon does not imply endorsement by Regulatory Focus or RAPS.