After facing a 16-month sojourn during Covid to overcome a stunning CRL, little Enzyvant says it's back on track — expects marketing decision later this year
Back in late 2019, Enzyvant CEO Rachelle Jacques was shocked when the FDA issued a CRL for its therapy to treat ultra-rare cases of pediatric congenital athymia — a condition marked by extreme immune deficiency and an early death. Like a lot of older, bigger organizations, Jacques discovered that regulators were not eager to OK the CMC setup at Enzyvant, though she told me at the time that she believed that with no demands for new clinical data, she felt that it could all be resolved in “a few months.”
Today, Jacques is putting out word that Enzyvant has answered all the FDA’s challenges and put their resubmitted BLA for RVT-802 back on track for a PDUFA deadline of October 8 — close to 2 years after the stunning setback.
So what happened?
First, she and her team decided to prepare carefully to set up their Type A meeting in March of last year to get a detailed read on expectations.
“Really, when coming out of that meeting then the work began,” says the CEO. “The meeting was meant to be face-to-face but it was about a week or so after the shutdown started. We were lucky that the agency pivoted that to a virtual meeting. It hit all the marks and we got what we needed out of it.”
So, what, exactly, did they have to do to resolve the CRL?
“We did need to do some facilities construction and we did that during Covid,” says Jacques. “That’s something that we didn’t anticipate immediately when we had the letter in our hands, but certainly as we talked with the agency that was very clear that was a solution. A lot of the other work was really just documenting at a very granular level some of the processes and so on as well as some additional studies we did to supplement the data we provided on the overall manufacturing process — and those were long lead time items as well.”
Enzyvant was a creation of Roivant involved in the big Sumitovant buyout and initially was in the lead position for delivering the first approval for Vivek Ramaswamy’s group. That first timer honor went to Myovant as Enzyvant was forced back in the line. Jacques isn’t saying how big the team is at Enzyvant after working through the resubmission, only going with “under 50” to indicate size.
Safe to say, it’s tiny.
The biotech has sidelined its one other drug program, an enzyme replacement therapy, shopping it to any bidder interested in taking it on. And the CEO says the team will stay 100% focused on a therapy that covers some 20 infants a year. Pricing hasn’t been established yet, she adds, but noting the complexity and trauma associated with treating these infants with no currently approved drug, it doesn’t sound like it will break ranks with a common industry formula to go higher as populations shrink.
Next steps include a look beyond America’s borders.