Global Blood Therapeutics Up On Expanded Use Plans For Sickle Cell Treatment

Shares in Global Blood Therapeutics (GBT) rose 5% in Monday’s extended trading after the company announced plans to expand the potential use of Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in children ages 4 to 11 years. The decision followed a Type B meeting with the U.S. Food and Drug Administration (FDA).

Oxbryta is the first and only therapy that directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The oral, once-daily therapy was approved in the United States in November to treat SCD in adults and adolescents ages 12 years and older.

“By mitigating red blood cell sickling and destruction early in life, we hope to positively impact the course of the disease,” said Ted W. Love, M.D., CEO of GBT. “We are pleased to have agreed with the FDA on a potential path forward to expand the use of Oxbryta in younger children, and we look forward to submitting a New Drug Application by the middle of next year.”

GBT plans to submit this New Drug Application (NDA) for Oxbryta under the FDA’s accelerated approval pathway, and will include data from the ongoing Phase 2a HOPE-KIDS 1 study. The FDA’s accelerated approval pathway can allow for earlier approval of drugs that treat serious conditions and that fill an unmet medical need.

“We’ve got a significant amount of data that really reinforces that the drug has a very similar profile for both safety and efficacy in this age group,” Love told Reuters on June 8.

Thanks to a strong Oxbryta launch, analysts currently have a bullish Strong Buy consensus on GBT stock, with 10 recent buy ratings vs 3 holds. With shares down 17% year-to-date, the $109 average analyst price target indicates upside potential of 66%. (See GBT stock analysis on TipRanks).

“In its first full quarter on the market Oxbryta blew away Street estimates ($14.1M vs. $4M for consensus) and posted encouraging metrics that exceeded our expectations at this early stage; a clear positive for the company” wrote JP Morgan’s Matthew Holt in May.