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

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

More from New Products

Novartis Upbeat Even With Entresto Expiry Looming

 
• By 

Swiss major confident that new oncology drugs will fill revenue gap.

J&J Delivers In Q2 Despite Stelara Drop, Raising Guidance

 

Stelara revenues declined 42.7% in the quarter due to biosimilars, but innovative medicines grew 4.9%, surpassing $15bn for the quarter. Although pharma tariffs could be coming, the firm said the impact continues to be hard to predict.

Sun’s Alopecia Treatment Debuts In US - Can It Make Up For Lost Time?

 

Sun’s Leqselvi goes up against Lilly's Olumiant and Pfizer's Litfulo, which currently has the broadest label, in the US alopecia market. Can it shake things up early, amid growing activity in the space with several new mechanisms of action being investigated?

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

 
• By 

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.

More from Scrip

Actuate Is Looking For A Buyer To Advance Pancreatic Cancer Candidate

 

After presenting encouraging Phase II data on the GSK-3 inhibitor elraglusib at ASCO, Actuate Therapeutics is hoping to hold a pre-NDA meeting with the US FDA.

Novartis Upbeat Even With Entresto Expiry Looming

 
• By 

Swiss major confident that new oncology drugs will fill revenue gap.

In Brief: Kezar Plots Lupus Revival For Zetomipzomib As FDA Lifts Hold On Hepatitis Trial

 
• By 

The go-ahead to resume a trial for its only drug candidate, zetomipzomib, in hepatitis could give Kezar a glimmer of hope of reviving another halted study in lupus nephritis.