1. Pink Sheet
  2. >>Agency Leadership

PTC Adds To Management Team In Sign Of Progress Beyond Setbacks

 
• By Lisa LaMotta

The New Jersey-based biotech has not lost its optimism after a setback in Europe for its lead product and is showing growth in other areas.

Despite some delays to its lead program in Duchenne muscular dystrophy, ultra-rare disease company PTC Therapeutics Inc. is forging ahead with other projects and is ready to show investors that it’s not a one-trick pony.

The announcement that it has initiated a Phase I trial of its second drug candidate coincided with the hiring of...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet 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 Agency Leadership

Early Support For EMA’s Trial Of US-Style Standards In Non-Clinical Reviews

 
• By Vibha Sharma

An ongoing proof-of-concept study by the European Medicines Agency testing the use of US-style standards for submitting non-clinical raw data in marketing applications has received positive feedback from both industry and regulatory assessors so far.

US FDA’s Tough Talk On Talc: Regulating In An Echo Chamber

 
• By Michael McCaughan

An FDA “expert roundtable” on talc produced a new talking point on a potential drug safety risk, but the basis of that claim is difficult to determine.

CAR-T REMS Removal Should Boost Access, But Long-Term Follow-Up, Black Box Remain

 
• By Sarah Karlin-Smith

The REMS removal and labeling changes should open up CAR-T treatment to many more US patients, although other regulatory hurdles and safety language that experts had wanted removed still stand.

PDUFA VIII: Cutting Perceived Conflict Of Interest May Be A Theme

 
• By Derrick Gingery

Ongoing questions about user fees exerting too much influence over the FDA could spawn a shift in the PDUFA structure when talks begin later this year.

More from Pink Sheet

COVID-19 mRNA Vaccine Labels Now Quantify Myocarditis Risk, But ACIP Wants Other Changes

 
• By Sue Sutter

New members of the CDC’s Advisory Committee on Immunization Practices suggested concerns beyond myocarditis, questioning whether test-negative observational studies are giving an incorrect picture of effectiveness.

Medicare’s Combo Products Negotiation Plan Conflicts With US FDA Standards, Firms Argue

 
• By Cathy Kelly

The plan could curtail a period of pricing flexibility for newer subcutaneous versions of important oncology drugs.

EU Pharma Overhaul May Reshape Aspects Of Drug Promotion And HCP Partnerships

 
• By Eliza Slawther

Lawyers explain how changes to rules around drug advertising and promotional activities under the EU pharma reform package could impact companies operating in the bloc, with one legal expert advising companies to “monitor developments closely.”