1. Scrip
  2. >>New Products

BioMarin Expects 30% Markdown For Orphan Pediatric Drug Brineura

 
• By Emily Hayes

Fast US approval of ultra-rare pediatric disease drug Brineura (cerliponase alfa), with an annual list price of $702,000, is a victory for the company, but approval for restricted pediatric population compared to Europe raised questions.

BioMarin Pharmaceutical Inc. gave its Brineura (cerliponase alfa) a high sticker price of $702,000 per year for treatment of a life-threatening, ultra-rare pediatric brain condition, but the company expects for most patients it will cost $486,000 – almost 31% less – upon launch in June.

See Related Story

For an analysis of what other recent high-priced launches mean for Brineura, see Few Clouds On The High-Priced, Ultra-Orphan Drug Horizon.

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

In Brief: Nanoscope Ploughs Forward With FDA Application For Retinitis Pigmentosa Gene Therapy

 
• By Manas Mishra

Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.

Pipeline Watch: Ten Approvals And Two 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.

Moderna’s Spikevax Wins Pediatric Approval, But With Narrower Label

 
• By Alaric DeArment

The approval for young children, consistent with the FDA’s new COVID-19 vaccine policy, restricts Spikevax to those with conditions putting them at higher risk.

Bayer Bags First Global Okay For Hot Flashes Therapy

 
• By Kevin Grogan

Lynkuet will compete with Astellas's Veozah/Veoza

More from Scrip

Q2 Korea Biotech Roundup: Licensing Deals End, Trials Progress, IPOs Recover

 
• By Jung Won Shin

Aside from several sizable global out-licensing deals, South Korean bioventures saw a cancellation of other alliances, updated their pipelines with losses and gains and reported clinical trial progress in the second quarter, while domestic IPO sentiment also improved.

Bayer’s Kerendia Gains Blockbuster Indication For Heart Failure

 
• By Joseph Haas

New US approval in heart failure with preserved or mildly reduced ejection fraction should add to Kerendia’s earning power on top of chronic kidney disease indication.

Takeda Prepares Ground For Oveporexton In Narcolepsy

 
• By Alaric DeArment

The drugmaker announced positive results from two Phase III trials for the drug in narcolepsy type 1 that analysts said bode well for the OX2R agonist class as a whole.