1. Pink Sheet

Why The Right-To-Try Law Is Not Right For Some Biotech Companies

 
• By Sue Sutter

Two CEOs explain why their rare disease companies will not grant preapproval access to investigational drugs under new federal law; Alnylam sees no need for framework beyond FDA’s expanded access pathway, while Sarepta is focused on the ‘grand goal’ of getting drugs studied and approved for the patient community.

The federal right-to-try law was a prime topic for conversation at the recent 2018 BIO International Convention in Boston, coming just days after President Trump signed the measure that creates a new pathway for preapproval access to investigational medicines.

The Pink Sheet spoke with the CEOs of Alnylam Pharmaceuticals Inc. and Sarepta Therapeutics Inc., two biotech companies...

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 Rare Diseases

More from Pink Sheet

EMA Explores Supporting Drug Development For High Unmet Need Disorder Scleroderma

 
• By Neena Brizmohun

While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.

EMA Nod For First MASH Drug Rezdiffra, Cell Therapy Zemcelpro And 11 Others

 
• By Eliza Slawther

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

BIO Notebook: Policy Uncertainty Constraining Dealmaking

 
• By Mandy Jackson and Joseph Haas

Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.