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In a comeback, Portola scores FDA accelerated approval for once-rejected Andexxa

Somehow staying on the good side of the FDA, Portola $PTLA has scored an elusive regulatory win for their once-rejected drug andexanet alfa.

The anti-anticoagulant — to be marketed as Andexxa — won the OK via the accelerated approval pathway, meaning that Portola will have to run a post-marketing trial to demonstrate an ability to control bleeding in patients.  The approval was based on data from two Phase III studies, conducted among healthy volunteers, that showed Andexxa could reverse anticoagulant activity of the Factor Xa inhibitors Xarelto (rivaroxaban) and Eliquis (apixaban). The drug also comes with a black box warning and is indicated for use when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.

Bill Lis

Portola has fought long and hard for this win. It first submitted an NDA for Andexxa late 2015, only to be caught off guard with an FDA rejection. At the time, the South San Francisco biotech said the agency was looking for more information on manufacturing and how the antidote works in other blood thinning drugs, such as edoxaban (Savaysa) and enoxaparin (Lovenox).

The team filed again in August 2017, exactly a year after it got the complete response letter. Regulators have since pushed back the action date from February to May 4, and Portola hinted in a recent earnings call that there could be further delays as the FDA requested further data, sending stock down. But nothing came out of that warning, and Portola spread word about the OK late Thursday — right before the original action date.

A spokesperson told me that the CRL Portola received in 2016 “focused almost solely on manufacturing concerns, which Portola addressed.”

“Andexxa is a breakthrough product, and as such, clinical was much faster than manufacturing,” they wrote. “In other words, at that time, manufacturing had to catch up with clinical.”

In a lengthy press release, Portola did not elaborate on how it resolved the FDA’s previous concerns, choosing instead to highlight the market potential of its reversal agent in an era when Factor Xa inhibitors have become increasing popular. One of them is Portola’s own Bevyxxa, whose approval raised some eyebrows since it barely failed a pivotal trial. European regulators gave it a thumbs down.

“We are proud that Andexxa is a first-in-class medicine discovered in our labs,” said CEO Bill Lis. “We remain committed to our scientific leadership in the fields of thrombosis and hematologic cancers.”

The company now plans to launch an early supply program of “Generation 1” Andexxa in June, with broader commercial launch anticipated in early 2019, assuming the FDA greenlights the manufacturing process of its Generation 2 product.

Meanwhile, the post-marketing trial is slated to begin in 2019 and be reported in 2023.

Portola shares edged up 3% in pre-market trading. According to EvaluatePharmaAndexxa carries a 2022 sellside revenue forecast of $749m.


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