Two of Harvard’s top FDA policy experts, Aaron Kesselheim and Jerry Avorn, have expressed some deep concerns about the agency’s approval of eteplirsen, OK’d to be sold as Exondys 51 for $300,000-plus a year, and the implications for further showdowns in the near future as more developers look to test the boundaries on the agency’s standards.
Writing online in the Journal of the American Medical Association, the two reviewed the campaign used to back eteplirsen, winning an OK with scant evidence of success in producing dystrophin – contrary to what Sarepta had claimed – a problematic reliance on historical comparisons, and the unanimous opposition of the main reviewers, including senior officials who appealed Janet Woodcock’s lone insistence on an accelerated approval.
