Drugmaker Agios Pharmaceuticals (NASDAQ: AGIO) is positioning to win the FDA's approval of its mitapivat as a treatment for sickle cell disease sooner than investors may have been expecting.

That's the overarching takeaway from this morning's press release anyway. As it explains, Agios' recent meeting with the U.S. Food and Drug Administration laid the groundwork for the "submission of a proposal for a confirmatory clinical trial to support U.S. accelerated approval of mitapivat." The company's already submitted the proposal for this abbreviated trial, in fact, and expects to submit its request for accelerated full approval to the FDA within the next few months.

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Agios Pharmaceuticals' oral pyruvate kinase (PK) activator mitapivat is proving to be a capable multipurpose therapy. Already approved to treat several forms of anemia, the drug has also performed well in the company's so-called RISE UP trials as a treatment for sickle cell disease ... a relatively rare disease with a limited number of effective treatment options. The drug performed so well, in fact, that the company's looking for somewhat special regulatory treatment of mitapivat when used for this particular purpose.

The FDA's accelerated drug approval process is designed to bring more urgently needed treatments to the market sooner than they'd normally become available. Although this pathway doesn't guarantee approval, it does underscore the FDA's perceived need for more therapy options for a particular disease.

In light of the FDA's recommendation for satisfying the regulator's usual drug-approval requirements with only an abbreviated "confirmatory trial," as of 12:05 p.m. ET Tuesday shares of Agios Pharmaceuticals are up 21.7%.

The market's knee-jerk response is understandable. This is good news, particularly for a drug that's already approved for other, similar purposes without any major safety concerns. The underserved sickle cell disease therapy market currently worth a little less than $4 billion per year could grow to more than $14 billion per year by 2034, according to an outlook from Precedence Research. It just needs more effective treatments. Mitapivat could be one of them, expanding the market's size simply by working well enough to prescribe more often.

Just bear in mind Agios is a young pharmaceutical outfit that's still losing a significant amount of money with a fairly small drug portfolio. Indeed, for perspective, it lost $413 million last year on revenue of $54 million.

Still, this isn't anything particularly unusual for the pharma business. If you can stomach the risk and inevitable volatility, the fact that shares are still well under recent peak highs even after today's big jump means there's room and reason for this ticker to continue moving higher.

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James Brumley has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.

Why Agios Pharmaceuticals Stock Is Up More Than 21% Today was originally published by The Motley Fool