1. Scrip
  2. >>Advanced Therapies
  3. >>Gene Therapies

Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US

Conditional Approval Granted In EU In August

 
• By Mandy Jackson

BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.

Round 2 neon sign with a Brick Wall Background
BioMarin is making a second attempt at FDA approval • Source: Shutterstock

BioMarin Pharmaceutical Inc., despite multiple setbacks, easily maintained its lead in the race to bring a hemophilia A gene therapy to market in the US with the resubmission of its biologic license application for Roctavian (valoctocogene roxaparvovec), kicking off a review process that could result in US Food and Drug Administration approval by mid-2023.

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 Gene Therapies

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
• By Alaric DeArment

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

Ferring’s Faith In Bladder Cancer Gene Therapy Starts To Pay Off

 
• By Kevin Grogan

Adstiladrin sales hit €70m in first full year on the market

What Is Fuelling Cell And Gene Therapies In India?

 
• By Vibha Ravi

Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?

Lilly Licenses Sangamo’s Capsid Technology For CNS Gene Therapy

 
• By Alaric DeArment

Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.

More from Advanced Therapies

Cartesian Maps Out CAR-T Strategy With Simpler Logistics In Mind

 
• By Alaric DeArment

The company announced positive Phase IIb data for Descartes-08 in myasthenia gravis, with especially strong efficacy among patients without prior biologic treatment.

What Is Fuelling Cell And Gene Therapies In India?

 
• By Vibha Ravi

Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?

Lilly Licenses Sangamo’s Capsid Technology For CNS Gene Therapy

 
• By Alaric DeArment

Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.