1. Scrip
  2. >>New Products

Its Gene Therapy Roctavian Finally Approved With $2.9m Price, BioMarin Aims To Prove Doubters Wrong

FDA Label Muddies Efficacy Claims

 
• By Andrew McConaghie

BioMarin has finally brought its hemophilia A gene therapy Roctavian market and is determined prove naysayers wrong, but must work with its limitations and a confusing US label.

Brisbane, CA, USA - Mar 1, 2020: The BioMarin logo seen at American biotechnology company BioMarin Pharmaceutical Inc.'s office in Brisbane, California.
• Source: Shutterstock

BioMarin has gained US approval for Roctavian, the first ever hemophilia A gene therapy, and the company is confident it can prove doubters wrong about how popular the one-time treatment will be with patients and payers. 

However, the high $2.9m price tag on Roctavian (valoctocogene roxaparvovec), a money-back guarantee for payers, and a lack of clarity...

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

Novartis Upbeat Even With Entresto Expiry Looming

 
• By Kevin Grogan

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

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

 
• By Jessica Merrill

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?

 
• By Anju Ghangurde

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 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.

More from Scrip

Novartis Chief Warns Of Fewer Launches And Drug Withdrawals In Europe

 
• By Kevin Grogan

Reimbursement rules are disincentivizing innovation, according to Vas Narasimhan.

Executives On The Move: Five New CEOs Among This Week’s Changes

Recent moves in the industry include changes at the top at Ascentage Pharma, Inventiva and Brenig Therapeutics, plus five companies get new CFOs.

AI-First, Big Pharma, Chinese Firms In Race - What’s Ahead For PRMT5 Inhibitors

 
• By Vibha Ravi

As AI-driven firms like Insilico, big pharma like J&J, Amgen, GSK and AstraZeneca and Chinese and Indian firms like BeOne and Dr. Reddy’s respectively advance PRMT5 inhibitor candidates, what’s driving interest, what could lead to the first global approval and what are the challenges ahead?