New FDA expanded access guidelines spell trouble for Sarepta
pharmafile | June 2, 2016 | News story | Medical Communications, Research and Development, Sales and Marketing | Duchenne Muscular Dystrophy, Eteplirsen, FDA, compassionate grounds, sarepta
New guidelines intended to streamline the process of expanded access to medicines by the US Food and Drug Administration (FDA) has led to shares falling almost 20% at Sarepta Therapeutics (NASDAQ: SPRT), who awaits an FDA decision on their Duchenne muscular dystrophy drug.
The FDA measures will attempt to reduce procedural burdens on physicians seeking to apply for patients to access drugs on “compassionate grounds”.
In the case of Sarepta, it seems investors believe these guidelines could have a damaging knock-on effect. The FDA delayed the announcement of its decision on whether to approve eteplirsen but a panel at the regulator expressed misgivings about the drug. A full approval of the drug seems unlikely to occur at this stage.
However, the streamlining of the expanded access to medicines process could mean that eteplirsen still gets to patients who vitally need it, with the caveat that Sarepta will only be able to recover the manufacturing costs of the drug under this initiative. The FDA states that a company “may recover only its direct costs associated with making the drug available to the patient… the sponsor may not charge for indirect costs, including administrating costs associated with providing an investigational drug.”
While patients may still benefit from the drug, by rejecting the drug the FDA could deny Sarepta the opportunity to set their own price on eteplirsen. Shares had initially rallied at the firm after the delay in the FDA’s decision on its new drug application, but it is clear that there are fears that this new process could form the basis of a complete response letter to be sent to Sarepta rejecting the drug.
Sean Murray
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