UPDATED: Apellis bags FDA nod for Soliris challenger with a different pathway to PNH — but can it slay the giant?
With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.
The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).
In pivotal Phase III data on which the agency based its review, Empaveli bested Soliris in a head-to-head test in terms of hemoglobin production with a relative increase of 3.9 g/dL (p<0.0001), and some favorable trend lines in secondary endpoints such as transfusions and fatigue.
Empaveli will come with the same black box warning label as Soliris, restricting use under a risk evaluation and mitigation strategy for meningococcal infections. The therapy was approved for patients regardless of prior therapies, meaning its use won’t be restricted to former C5 patients alone.
On a call with analysts Monday, Apellis said Empaveli would come with an average annual wholesale acquisition cost of $458,000. That’s “at parity” with Ultomiris and a discount on Soliris, the company said.
Apellis thinks that a big bump in hemoglobin levels could help it immediately capture about a third of the existing PNH patients who have a low and falling hemoglobin count that is currently underserved by the C5 drugs, CEO Cedric Francois told Endpoints News prior to the approval.
“If the Phase III study did one thing it is making it very clear that by controlling extravascular hemolysis, you can elevate the standard of care in these patients,” Francois said.
The key to Apellis’ pitch is C3, which it believes can offer a longer-lasting solution for hemoglobin production as C5 often doesn’t boost levels for more than a few days, the team said. With an approval in hand, Apellis said it plans to target those 500 or so US patients with the lowest hemoglobin counts but thinks Empaveli could have a meaningful uptake across the entire PNH spectrum.
The company has already built up a sales team that has been doing some of the groundwork on physician outreach in the lead-up to approval. It’s a small group of PNH specialists, and Apellis thinks it’s found a workable, hybrid digital/in-person education campaign to tell doctors about the drug’s benefits.
But beating Alexion — and soon to be AstraZeneca — won’t be an easy task by any means. Alexion’s entire franchise was built around building Soliris into a blockbuster, and the company has been busy amid a gung-ho switching campaign to follow-up drug Ultomiris.
AstraZeneca announced its intent to buy Alexion late last year for a cool $39 billion. The rare disease specialist will import its expansive portfolio of C5 drugs, which sport indications beyond PNH.
This story will be updated.