1. Scrip
  2. >>New Products

Few Clouds On High-Priced, Ultra-Orphan Drug Horizon

 
• By Mary Jo Laffler and Emily Hayes

With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.

Money Wave

Pricing has never really been an issue for rare disease drugs, given the low exposure payers have to the therapies and the significant benefits usually offered. But with ultra-orphan price tags ticking higher and higher and drug pricing a simmering political controversy in the US, it is worth checking on the experience of recent launches as BioMarin Pharmaceutical Inc. introduces its Batten disease therapy Brineura (cerliponase alfa) at just over $700,000.

See Related Story

For details of the Brineura approval, see BioMarin Expects 30% Markdown For Orphan Pediatric Drug Brineura.

Two antisense oligonucleotide therapies approved last year for different neurodegenerative conditions, Biogen/Ionis Pharmaceuticals Inc.'s Spinraza (nusinersen) for spinal...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from New Products

Pipeline Watch: Ten Approvals And Seventeen Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Defying Gravity: Five Drugs That Surpassed Their Sales Forecasts

 
• By Edwin Elmhirst

Many assets outperform their pre-launch sales predictions despite the frequent overestimation of forecasts within the industry. In this article, Scrip highlights several such assets and the factors that influenced their predictions.

Gilead’s Yeztugo Gets US Approval For Twice-Yearly PrEP

 
• By Alexandra Shimmings

The first twice-yearly option for HIV prevention has been approved by the US Food and Drug Administration in an advance that “brings us closer than ever to ending the HIV epidemic,” said CEO Daniel O’Day.

Pipeline Watch: Eight Approvals And Ten Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

More from Scrip

What Incyte Investors Expect From New CEO Bill Meury

 
• By Andrew McConaghie

Fresh from having sold Anthos to Novartis, the new CEO is expected to update the oncology and dermatology-focused firm's strategy.

Bayer Buoyant After EU Nod For Twice-Yearly Eylea

 
• By Kevin Grogan

Green light follows a recent rejection by the US FDA to partner Regeneron's request for an extension to the dosing interval.

Executives On The Move: New CEO At Atalanta Therapeutics

Recent moves in the industry include C-suite changes at Atalanta Therapeutics and Climb Bio, plus seven companies get new CMOs.