Breakthrough Sickle Cell Disease Treatment Is on the Way

By Chris Lange Updated Jan 6, 2:45AM EST · Published Sep 5, 10:20AM EDT

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Breakthrough Sickle Cell Disease Treatment Is on the Way — © Dr_Microbe / Getty Images

Global Blood Therapeutics Inc. (NASDAQ: GBT) shares jumped on Thursday after the company announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA).

Specifically, this NDA is for voxelotor for the treatment of sickle cell disease (SCD). If approved, voxelotor would be the first therapy available to patients that targets hemoglobin polymerization, the root cause of SCD damage.

The FDA granted Priority Review for the NDA for voxelotor, which provides for a six-month review, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2020. The FDA also indicated in the NDA filing acceptance notification letter that it is not planning to hold an advisory committee meeting to discuss the application for voxelotor.

The NDA for voxelotor is supported by data from the multinational Phase 3 Hope Study of voxelotor in patients ages 12 and older with SCD. Previously, voxelotor has been granted FDA Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD.

Ted W. Love, M.D., president and CEO of GBT, commented:

The FDA’s acceptance of our NDA for voxelotor under Priority Review is a major milestone in the development of this investigational therapy and further illustrates the significance the Agency places on getting important and innovative treatments to individuals living with SCD as quickly as possible,” “We look forward to working with the FDA during this process, with the goal of potentially changing the treatment paradigm for SCD.

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Janney Capital was quick to weigh in on GBT’s win:

We believe the smooth NDA acceptance by the FDA has been expected, but we see the priority review, with the agency confirming that there will be no AdCom, as a strong positive that suggests the FDA’s comfort towards the MOA of voxelotor and the overall clinical profile of the drug candidate. With a PDUFA date of 2/26/2020 and active ongoing commercial preparations by the company, a U.S. launch could be possible in 1Q20. And upon the FDA approval, the Rare Pediatric Disease designation for voxelotor will bring GBT with a rare pediatric disease priority review voucher (PRV), which in the past has been sold for as high as $350M. Based on our most recent discussion with management, we believe confirmation study in pediatric patients, using transcranial Doppler (TCD) as the primary efficacy endpoint, is on track to be initiated in 4Q19 and the announcement of study initiation should be the next major milestone.

Shares of GBT traded up over 13% on Thursday to $53.77, in a 52-week range of $30.15 to $64.94. The consensus price target is $91.85.

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About the Author Chris Lange →

Chris Lange is a writer for 24/7 Wall St., based in Houston. He has covered financial markets over the past decade with an emphasis on healthcare, tech, and IPOs. During this time, he has published thousands of articles with insightful analysis across these complex fields. Currently, Lange's focus is on military and geopolitical topics.

Lange's work has been quoted or mentioned in Forbes, The New York Times, Business Insider, USA Today, MSN, Yahoo, The Verge, Vice, The Intelligencer, Quartz, Nasdaq, The Motley Fool, Fox Business, International Business Times, The Street, Seeking Alpha, Barron’s, Benzinga, and many other major publications.

A graduate of Southwestern University in Georgetown, Texas, Lange majored in business with a particular focus on investments. He has previous experience in the banking industry and startups.