Slammed by FDA rejection, Aquestive vows to refile by end of year
Aquestive Therapeutics is still reeling from a precipitous drop in its stock price after the FDA rejected its oral film for seizure control.
Libervant, a buccal film formulation of the generic drug diazepam, would have been the biotech’s second commercial treatment for epilepsy following Sympazan (clobazam), which is approved specifically for Lennox-Gastaut syndrome.
According to a release out Friday, regulators are mainly worried about pharmacokinetics, citing in the complete response letter a study where “certain weight groups showed a lower drug exposure level than desired.”
While Aquestive said they are clear on safety, clinical pharmacology or manufacturing, regulators did also take issue with a small number of protocol deviations in blood draws in one of the studies. It’s unclear how big that concern looms.
Next steps include providing more information on PK modeling to convince the FDA that the issue can be solved by dose adjustments, but no additional studies.
Still, it is a notable setback for a company that’s built its reputation on a technology platform to redesign and deliver medications through film.
Offering already available drugs in a different form, the reasoning goes, allows them to address different patient populations.
“Epilepsy patients have been underserved for some time with little choice beyond device-based products such as rectally administered gels and nasal sprays and Libervant represents a meaningful and improved therapy for patients who can’t or won’t use the alternatives,” CEO Keith Kendall said in a statement.
Similarly with Exservan, the film version of riluzole that passed FDA muster last November, Aquestive was targeting amyotrophy lateral sclerosis patients who have trouble swallowing pills.
Almost a year after the Exservan OK, though, Aquestive has yet to launch the drug.
For Libervant, the biotech says it will be requesting a Type A meeting with the FDA in the coming weeks and expects to refile an NDA by the end of the year.
That didn’t stop investors from sending the stock down 34% to $4.97 on Monday, which went down some more during the day.