Shire’s got a brand-new approval on its hands that could help it dominate the hereditary angioedema (HAE) market for years to come—and rack up blockbuster sales in the process.

Thursday, the FDA green-lighted Takhzyro for HAE prophylaxis in patients 12 and older. And with IP protection to 2032, orphan drug status, "impressive" efficacy data and a convenient dosing regimen for the newcomer, "today’s approval should position" Shire to "not only maintain but also significantly grow its dominant HAE franchise," Jefferies analyst David Steinberg wrote in a note to clients, adding that the drug could become "best-in-class." 

Regulators gave Takhzyro the go-ahead based on phase 3 data showing the drug, dosed every two weeks at 300 mg, could cut down the incidence of monthly attacks by 87%. Monthly dosing at both 150 mg and 300 mg spurred 76% and 73% reductions, respectively.

Of course, Takhzyro isn’t the first new HAE drug to post big numbers. It’ll compete against CSL’s Haegarda, which was approved last year despite Shire's best efforts to block it. That drug has also showed a median reduction in monthly attacks of between 89% and 95% versus placebo, Steinberg noted. But that’s where Takhzyro’s favorable dosing regimen comes in: Patients receive the Shire drug just once or twice a month, compared with twice a week for Haegarda.

All told, Steinberg thinks Takhzyro could "significantly expand the global HAE market" on the way to racking up $1.8 billion in peak sales—especially since about 45% of U.S. and EU patients are still undiagnosed, a figure that’s even higher in emerging markets. And that's good news for Takeda, which in May agreed to shell out a controversial $62 billion for the Dublin drugmaker.

First, though, Shire will have to get payers on board, and if Shire goes with a high price tag typical of rare disease fighters, that task could have its challenges. The company, though, has pointed out that other HAE products "should allow for better positioning with managed care and the reimbursement environment globally," Steinberg wrote.

With drugs such as Cinryze and Firazyr also within its portolio, Shire has "the ability to offer different price points and modalities that could cater to different segments of the market," he added.

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