FDA OKs CSL’s new HAE drug, already embroiled in a blockbuster fight with Shire

CSL Behring’s Bern, Switzerland manufacturing campus

CSL Behring has picked up an FDA OK to start marketing its new drug for rare cases of hereditary angioedema, which is already embroiled in a court fight triggered by its HAE rivals at Shire. And in doing so, the FDA has now crossed the line for all its new drug approvals of 2016, still just shy of the halfway mark to the year.

The agency’s approval for the C1 esterase inhibitor Haegarda came through Thursday evening, two months after Shire fired a preemptive legal shot in US District Court in Delaware claiming that CSL’s drug violates its new patent for its own C1 drug Cinryze. Shire — which saw its shares $SHPG drop 1.3% Friday morning — also markets Firazyr and Kalbitor for HAE, a franchise that CSL hopes to carve up with a drug that can be self-administered.

It comes with a clear endorsement from the FDA.

“The approval of Haegarda provides a new treatment option for adolescents and adults with hereditary angioedema,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research. “The subcutaneous formulation allows patients to administer the product at home to help prevent attacks.”

The drug was green lighted by regulators after investigators put it through a pivotal trial with 70 patients, demonstrating its superiority over a placebo in preventing attacks. It will now go into a small market with some 6,000 to 10,000 patients in the US.

Flemming Ornskov

CSL is an experienced player in the rare disease field, which Shire CEO Flemming Ornskov has colored in as its own primary focus. CSL markets Idelvion with a $500,000 wholesale price. Idelvion — one of the top 10 most expensive drugs on the planet — is a long-acting hemophilia B drug that is the first in its class to include the blood protein albumin.

Shire is fighting for time as it hustles along an FDA application for its new HAE drug lanadelumab after bagging stellar Phase III data just last month. Shire’s drug — obtained in a $6.5 billion deal to acquire Dyax — has also been designated a breakthrough drug. Some analysts believe it could be worth $2 billion in peak sales, unless CSL manages to make the most of its first mover advantage.

CSL’s drug reduced HAE attacks by 89% and 95% while Shire’s new drug came in right alongside with an 87% reduction. These are not head-to-head results, though, reflecting different ways to measure efficacy. Those fine points can make a big difference.

Law360 noted that Shire filed a lawsuit against CSL the same day it obtained the patent for Cinryze.

These patent fights have become the order of the day in biopharma. Amgen has been particularly aggressive in the courts, pushing an advantage it has over Regeneron and Sanofi in a scuffle over its PCSK9 drug patents. And Juno just had its case against Kite thrown out after the judge decided that the court didn’t have jurisdiction after determining there was no way to guarantee that Kite’s CAR-T would soon be approved, even though it faces a looming PDUFA date.

Now at the halfway mark in the year, the FDA’s action marks the 23rd new drug approved so far in 2017, 1 more than for all of 2016.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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