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...
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PharmaMar has voluntarily withdrawn its marketing authorization application for Aplidin to treat multiple myeloma in the EU – almost a decade after it first filed the drug for EU approval and following years of court proceedings.
The European Medicines Agency’s human medicines committee, the CHMP, has recommended 14 new medicines for pan-EU approval this month, including five orphan medicines.
The European Medicines Agency says that Eli Lilly's Alzheimer's disease drug should be approved for use in the EU, now that it has re-examined the negative opinion it previously adopted.
Bristol Myers Squibb’s Opdivo and Merck's Welireg also could benefit from the delay in Medicare price negotiation process eligibility.
The US FDA says its release of complete response letters for products subsequently approved by the agency is just a first step toward releasing rejection letters when they are issued, assuming legal issues can be resolved.
The FDA's recent announcement of plans to expand unannounced inspections at ex-US facilities should put firms on advance notice that their next foreign facility inspection may not be preannounced, Ivy Sweeney, acting head of the agency's drug inspections office said.
New US FDA drugs center director George Tidmarsh’s data-centric comments at a Reagan-Udall Foundation meeting on unapproved fluoride products could suggest patients’ and caregivers’ experiences will carry less weight in benefit-risk decisions under his leadership.
