1. Scrip
  2. >>Drug Pricing

Stock Watch: Advanced Therapies Deliver Fourth-Quarter Flop

Despite Obvious Clinical Benefits, Successful Commercialization Remains Elusive

 
• By Andy Smith

As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.

Stock Watch Image, Andy Smith
ANDY SMITH OFFERS A LIFE SCIENCE INVESTOR'S PERSPECTIVE ON BIOPHARMA BUSINESS

BioMarin Pharmaceutical Inc.’s fourth-quarter and full-year 2023 financial results announcement prompted a modest outperformance by its stock compared with the NASDAQ Biotech Index’s (NBI’s) 1% increase the day after BioMarin’s post-market announcement. Analysts and investors were focused on the majority of BioMarin’s commercial operations and sales of its advanced gene therapy product – Roctavian (valoctocogene roxaparvovec) for hemophilia A – seemed largely forgotten despite growing by nearly 240% on the third quarter of 2023. This was because the percentage of BioMarin’s fourth-quarter total sales contributed by Roctavian was so low that it looked more like a highly significant p-value.

Following Roctavian’s approval at the end of June 2023, BioMarin’s fourth-quarter commentary on it was limited to the “build-out of...

More from Drug Pricing

BIO Notebook: MFN Pricing, Next-Gen Obesity R&D, FDA’s Rare Disease Hub & Reaction To Review Program

 
• By Sarah Karlin-Smith, Mandy Jackson, and Alaric DeArment

Highlights from Day 3 of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.

Stock Watch: Generics Firms Trapped Between US Tariffs And European Austerity

 
• By Andy Smith

Successive annual reductions in European drug prices following the pandemic are affecting the global sales of generic pharmaceutical companies. Additionally, the imposition of US import tariffs may result in generic drug supply becoming unprofitable.

UroGen Gets A First-Ever Bladder Cancer Approval Despite US FDA AdComm ‘No’ Vote

 
• By Joseph Haas

UroGen’s Zusduri was approved despite an advisory committee vote recommending against it in May. It is the first approved drug for a type of recurring bladder cancer.

Europe’s Drugmakers Lament EU Pharma Package

 
• By Kevin Grogan

EFPIA says reforms make the continent less attractive for innovative firms.

More from Scrip

Shigella Vaccine Pipeline And Why It Matters

 
• By Vibha Ravi

A GSK-Bharat Biotech deal on a shigella vaccine candidate has cast a spotlight on the disease which is the second leading cause of diarrheal deaths in children below five. Scrip looks at data from Pharmaprojects, WHO and other sources to highlight why a vaccine matters and what pharma is doing

Quick Listen: Scrip’s Five Must-Know Things

 
• By Ian Haydock

In this week's episode: US policies in spotlight at BIO; Lilly’s Verve acquisition; UroGen’s bladder cancer approval; Ireland’s biopharma talent; and Korea proposes new AI R&D project.

Asia Deal Watch: SunRock, Escugen Collaborate On CCR9-Targeted ADC

 
• By Joseph Haas, Lisa Takagi, Sushmita Panda, and Xu Hu

Axcelead/Lotte Biologics/Kanaph and NextCure/Simcere also unveil ADC-focused pacts, plus deals involving Avata/Oceanus, GSK/Bharat Biotech, Cullinan/Genrix and more.