The FDA has come up with a series of instructions spelling out new rules to biotechs on providing expanded access to experimental drugs, including a more streamlined approach for compassionate use aimed in part at making it easier for physicians to ask for an experimental drug for one of their patients. And the buzz among analysts immediately centered on speculation that the FDA was setting the stage to pull the rug out from under Sarepta ($SRPT) and formally reject its controversial Duchenne muscular dystrophy drug eteplirsen, sending its shares spiraling down 27%.
Spokespeople for the agency insisted that the update was long in the making and unrelated to any particular company, but the new guidances--which also included some added regulatory context on troublesome issues like the added threat of adverse events from expanded use--were seen as an interim step that was hurried up to take the some of the sting out of a CRL.
RBC’s Simos Simeonidis: “Making a drug like eteplirsen available through an expanded access process was already possible and had been considered for a while, but many questions lingered, including process, timing, cost, reimbursement. Today's guidance by FDA helped answer a number of these questions. Most importantly, it provided a simple roadmap by which the drug could get to patients quickly, thus relieving the significant political pressure being felt by the agency, without FDA having to approve the drug prior to the completion of randomized controlled trials.”
Baird’s Brian Skorney: “Although this guidance has been in the works for months, maybe years, we do find last week's PDUFA delay to be curious timing in light of this issuance. We’ve continuously said that this could be a good compromise for all involved.” Besides, he added, expanded access worked for Gentium and defibrotide and it could work well for Sarepta. Also, Sarepta needs more data for an approval, and this is one pathway to get to that next stage.
Joseph Schwartz at Leerink gave a nod to the update on charging for expanded access under compassionate use rules, which gives Sarepta a chance to regain its manufacturing costs. And he also pointed to rules that make it easier for companies, groups and individuals to apply for access, which would also help facilitate the process.
“All things considered,” Schwartz noted, “today’s guidance for industry appears to heighten the uncertainty of outcomes for eteplirsen but also potentially soften the blow for the patient community and SRPT, who has demonstrated the need for special treatment during the course of drug development.”
The FDA set up the latest round of speculation after it recently announced its second delay on making a formal marketing decision on eteplirsen. There’s no solid evidence that the agency was giving itself enough time to issue these rules before telling patients and families who have been fiercely lobbying for an approval that the agency will wait for more data before green-lighting the drug. But with Sarepta, in the absence of facts, guesswork becomes central to the debate over its future, driving the roller coaster ride in share prices that has been playing out for years.
In the meantime, the industry will be carefully examining the new rules to see how they could affect everyone in biotech. Drug developers have had a long, troubled relationship with compassionate use, leery that a patient getting a drug outside of a trial could experience an adverse event that would threaten their product and unhappy about the cost of providing drugs without profiting from it. Patients also face potentially sky-high out-of-pocket expenses, and payers don't want to cover a drug that may not work.
But despite the kickbacks, the demand from patients can be hard to ignore.
- here's the FDA announcement
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