1. Scrip
  2. >>Business
  3. >>Earnings

US FDA AdComm For Vertex’s Sickle Cell Gene Therapy ‘Not A Concern’

 
• By Ayisha Sharma

The US biotech revealed the US Food and Drug Administration would be holding an advisory committee meeting for exa-cel during its second quarter earnings, but analysts are unfazed by the news amid robust revenues and raised guidance.  

3d graphic of sickled blood cell
Sickle Cell Disease Affects Around 100,000 People In The US • Source: Shutterstock
Key Takeaways:

  • Sales of cystic fibrosis drug Trikafta rose due to uptake in the younger pediatric population.

  • Exa-cel could become the first gene therapy for SCD with an approval expected in December.

  • The firm’s Phase III pain program is gaining attention due to its blockbuster market potential.

Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s gene therapy candidate for inherited blood disorders, exagamglogene autotemcel (exa-cel), will be subject to a US advisory committee meeting,

The firm’s second-quarter revenues increased by 14% to $2

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 Earnings

BioNTech Aims To Diversify BNT323 Manufacturing As Filing Nears

 
• By Alaric DeArment

The company plans to expand manufacturing of the HER2-targeting ADC beyond China and plans to file for US FDA approval in 2025.

Lilly’s Ricks Urges Trump To Pick Tax Incentives Over Tariffs

 
• By Alaric DeArment

On Lilly’s Q1 earnings call, CEO David Ricks voiced concerns about the Trump Administration’s tariff policy, which he said could adversely affect Lilly and the industry as a whole.

Transient FY26 For Syngene But Momentum In China +1 Projects

 
• By Anju Ghangurde

As biopharma derisks its business with China ‘rebalancing’ strategies, Syngene could convert majority of such pilots into full program contracts amid a challenging FY2025. The CRDMO also expects business discussions pertaining to a newly acquired US biologics site to mature into commercial opportunities.

Biogen’s Manufacturing Sites, Revenue Sources Protect It From Big Tariff Impact

 
• By Mandy Jackson

Biogen expects little impact from existing tariffs and any that may apply if the US exemption of pharma products is lifted, since 75% of its manufacturing is in the US and 55% of sales are ex-US.

More from Business

Pharma Predicts Modest Impact From Tariffs, But It Depends On What Comes Next

 
• By Jessica Merrill

Drugmakers aren’t expecting a big financial hit from tariffs for now, but a report commissioned by PhRMA suggests the cost of pharma-sector tariffs could be steep.

OPPI’s Matai On Section 3(d) Of India’s Patent Regulations: Now’s The Time To Open Up

 
• By Anju Ghangurde

Anil Matai, director general, Organization of Pharmaceutical Producers of India, talks in this audio interview about the evolving intellectual property landscape in India post the 2024 amendments, including long-standing sticking points such as Section 3(d) of India’s patent regulations and innovator firms' experience of the Bolar provision. There’s also a "compelling reason" to consider regulatory data protection, he claims.

In Brief: Sanofi Gains €10bn From Opella Sale

 
• By Sushmita Panda

Sanofi sold a 50% stake in consumer health unit Opella for €10bn, backing its shift to prescription drugs and funding a €5bn buyback.