Global Blood Therapeutics, Inc. GBT announced that it plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (voxelotor) tablets. The company is seeking approval of the drug to treat hemolytic anemia in sickle cell disease (SCD) patients aged 12 years and older. It plans to submit the MAA by mid-2021.
In November 2019, the FDA approved Oxbryta as an oral, once-daily treatment for SCD in patients aged 12 years or above. Following this, the drug became the first and only therapy that directly inhibits sickle hemoglobin polymerization, a major cause for SCD. SCD is a chronic, inherited blood disorder that affects hemoglobin levels.
The planned MAA will include data from the phase III HOPE study and the phase II HOPE-KIDS 1 Study. Global Blood also intends to initiate an Early Access Program in Europe for patients and physicians who may need access to Oxbryta prior to potential marketing authorization.
Anemia and hemolysis are known to cause morbidity and mortality in SCD. Currently, there are no approved therapies in Europe to treat hemolytic anemia in SCD. Thus, the company intends to introduce a treatment for the disease.
Shares of Global Blood have declined 18.8% so far this year against the industry’s growth of 11.9%.
In June 2020, the companyannounced plans to expand the label of Oxbryta in the United States, which will include a new, age-appropriate formulation, for the treatment of SCD in children 4 to 11 years, under the FDA’s accelerated approval pathway. The NDA will include clinical data from the ongoing phase IIa HOPE-KIDS 1 study.
The company has another pipeline candidate, inclacumab, which is a novel, fully-human monoclonal antibody against P-selectin. The drug is being developed as a treatment for vaso-occlusive crises (“VOC”) in patients with SCD. The company is developing inclacumab with drug giant Roche Holding AG RHHBY.
Last year, the FDA also approved Novartis’ NVS Adakveo (crizanlizumab) to lower the frequency of VOCs or pain crises in SCD patients aged 16 years or above. bluebird bio BLUE) is also developing LentiGlobin for the treatment of VOC in patients with SCD.
Global Blood Therapeutics, Inc. Price
Global Blood Therapeutics, Inc. price | Global Blood Therapeutics, Inc. Quote
Global Blood currently has a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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