UPDATED: Actelion wins crucial FDA approval for next-gen lung disease drug Opsumit

Late on Friday the FDA came through with an approval for Actelion's pulmonary arterial hypertension (PAH) drug Opsumit (macitentan), its next-gen successor to the franchise drug Tracleer. And on Monday the Swiss company's shares shot up 7% on the news.

The approval comes as no great surprise. The company ($ATLN) had been steadily doing the rounds with regulators, armed with positive late-stage data. The lead-up to the Phase III read out, though, had precipitated considerable angst among some of the analysts covering the Swiss company. Actelion gets the bulk of its revenue from the PAH drug Tracleer and had to have a score here in advance of a loss of patent protection.

Opsumit will carry a boxed warning alerting patients and physicians that the drug should not be used in pregnant women because it can harm the developing fetus. But the FDA release made no mention of a required liver test in the warning, something some analysts felt would have blunted sales.

A spokesperson for the company told FierceBiotech that the new therapy will be priced similarly to Tracleer.

PAH drug development has been a busy field in recent years. Just days ago Bayer won an FDA approval for its lung disease drug Adempas (riociguat). United Therapeutics ($UTHR), though, recently picked up its second CRL from the FDA for an oral version of Remodulin. Gilead's ($GILD) Letairis--approved in 2007--has meanwhile been eating into Tracleer's market. 

Actelion is staying focused on R&D for PAH. The next drug in its pipeline is the Phase III treatment selexipag. Actelion licensed the ex-Japan rights to the drug from Nippon Shinyaku and expects to report late-stage data in mid-2014.

"Today's approval of Opsumit by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression," noted Actelion CEO Jean-Paul Clozel, who had to endure an unsuccessful shareholder revolt while he stayed the course on Opsumit's development. "Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit in the largest, longest and first-ever outcome study in PAH."

- here's the press release

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