Horizon notches blockbuster FDA approval for rare eye disease med Tepezza

FDA
An FDA advisory committee unanimously recommended Tepezza's approval back in December. (FDA)

Horizon Therapeutics is trying to shed its reputation as a primary care drugmaker and emerge with a brand-new life in rare diseases. Now, with an FDA approval for a hotly anticipated rare disease med in hand, Horizon's rebrand is well on its way—and to hear the company tell it, it'll report blockbuster sales soon enough with the help of a $14,900-per-vial list price.

The FDA Tuesday approved Horizon's Tepezza (teprotumumab) as a treatment for thyroid eye disease (TED), a rare autoimmune disorder that can lead to blurred or double vision. It previously had no approved therapies. Horizon said the drug will launch immediately at a list price of about $200,000 for a six-month course of treatment.

Tepezza is the first drug Horizon has shepherded through the FDA's Biologics License Application process, "a key step in our evolution to an innovation-focused biopharma company," CEO Tim Walbert said in a statement.

The FDA based its approval on phase 3 data showing 82.9% of Tepezza patients achieved a 2-mm or greater reduction in proptosis––or eye bulging––after 24 weeks compared with 9.5% of placebo patients. The drug also showed it could reduce clinical symptoms, ease double vision and improve patients' quality of life. 

Based on those results—and a U.S. market of 15,000 to 20,000 patients—Horizon's predicting blockbuster-level sales for its new med. TED patients typically seek treatment when they notice their eyes bulging in photos or that their eyes and eyelids constantly seem to be red and swollen. 

Horizon expects to launch a post-marketing safety and retreatment study for Tepezza pending discussions with the FDA on a possible trial's structure. 

RELATED: Horizon's eye drug teprotumumab wins FDA panel backing in unanimous vote

Horizon's rebrand follows years of controversy, when the company was known for buying older drugs and pushing through big price hikes. As pricing scrutiny mounted, it was among 22 companies barred from trade group PhRMA in 2017 after it instituted R&D spending requirements.

Since then, Horizon has raised its R&D spending to about $75 million during the first three quarters of 2019; during the same period of 2016, it spent about $36 million on R&D. The company shelled out $145 million to buy Tepezza's original developer, River Vision, in 2017 when the drug had just wrapped up successful phase 2 studies.

In an interview, Walbert said Horizon would lean on its 100-employee-strong commercial team to roll out Tepezza immediately.

Tepezza is now Horizon's second marketed biologic alongside gout med Krystexxa, which raked in more than $300 million in 2019. With both drugs estimated to hit blockbuster territory, Walbert said the company would be looking for acquisition targets to build its pipeline in nephrology, rheumatology and endocrinology.

"We have the right balance sheet, we have two billion-dollar medicines to drive growth, and that will allow the cash to flow to fund acquisitions that will help develop our portfolio," Walbert said.

As for what those acquisitions could look like? Horizon is angling for candidates that are post-proof of concept and in the phase 2 stage or later––bolstering its case as less of an R&D organization and one more focused on commercial execution. 

Horizon said it expects a "gradual uptake" for Tepezza this year with a sales estimate between $30 million and $40 million by year-end. 

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