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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 developing treatments for rare diseases, during the BIO meeting about...

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