What happened

A presentation at the J.P. Morgan Healthcare Conference in early January kindled excitement that Global Blood Therapeutics (NASDAQ:GBT) could be on the cusp of launching a blockbuster drug for sickle cell disease, a painful genetic disorder affecting hundreds of thousands of people worldwide. Word that management intends to talk about its new drug's application options with the Food and Drug Administration soon sent shares rocketing 16.7% higher in January, according to S&P Global Market Intelligence

So what

Global Blood Therapeutics has just one drug in its clinical-stage pipeline: voxelotor, an oral, once-daily treatment for sickle cell disease (SCD). Voxelotor helps prevent red blood cells from forming into their misshapen "sickle" look by boosting the amount of oxygen in hemoglobin. 

Ascending stacks of oral tablets sit on top of $100 bills.

IMAGE SOURCE: GETTY IMAGES.

In December, data was unveiled by management showing that a 1,500 mg daily dose of voxelotor increased hemoglobin at the 24-week mark by at least 1 gram per deciliter in 65% of patients. That was significantly better than the 33% of patients seeing that improvement following a placebo.

The company also said that the FDA has agreed to an accelerated pathway that could allow voxelotor to reach the market more quickly. A meeting is planned with regulators by the end of this quarter to discuss voxelotor's potential, according to a presentation at the J.P. Morgan conference last month.

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Now what

If the meeting goes well and voxelotor does eventually get a green light, then it could mark a major advance in treating SCD patients. SCD can cause painful vaso-occlusive crises that land patients in the hospital. On average, a sickle cell patient's life span is 20 to 30 years shorter than a healthy person's, and the cost of care for these patients is roughly $200,000 per year. Because over 100,000 patients in the U.S. alone have SCD and there's an undeniable need for new treatment options, hope is high that voxelotor has blockbuster potential.

However, there's no guarantee that voxelotor crosses the finish line. Its clinical trials are evaluating its ability to boost hemoglobin and brain blood flow, and that's not the primary end point typically used to support approvals in this indication. In the past, the focus has been on reducing vaso-occlusive crises. There's also the risk that the market for SCD treatment will be disrupted by gene therapies in development that are designed to restore normal hemoglobin production in a one-and-done dose. If gene therapy is successful, it could significantly reduce voxelotor's peak sales opportunity.