By Sean Marshall
This week it came to light that by the PBR Regulatory Affairs review board that the “clinic-stage bio-pharmaceutical firm Global Blood Therapeutics (GBT) has secured orphan drug designation from the US Food and Drug Administration (FDA) for GBT440 to treat sickle cell disease.”
That was a lot of information to take in but the basic concept behind it is that a new sickle cell fighting drug is in the clinical stages, making it one step closer to everyday people.
The drug titled GBT440 is meant to be an “oral, once-daily SCD therapy that works by increasing hemoglobin’s affinity for oxygen.” The drug itself would undue one of the biggest risks of sickle cell disease. It could in fact reduce the levels risks of stoke for anyone taking it.
It's also worth noting that Global Blood Therapeutics announced some of their testing plans. “the company is investigating the drug candidate in both healthy subjects and SCD patients in phase ½ clinical trial.” It was also explained that “the efficiency of GBT440 is being evaluated on polymerization of deoxy-hemoglobin, the mechanism of red blood cells sickling.”
All access to the results are to be available late 2016.
The PBR Regulatory Affairs report can be found here: http://regulatoryaffairs.pharmaceutical-business-review.com/news/fda-grants-orphan-drug-designation-to-gbt440-to-treat-sickle-cell-disease-040116-4766214
Other news this week included a Ministry of health announcement from Bahrain's health department that there is no shortage of morphine due to the treatment of sickle cell patients. The country of Bahrain is a small island n the Middle East. Like most of the world they to struggle with sickle cell disease.
Unfortunately there have been rumors of a shortage of morphine. This in turn lead many with sickle cell disease to go through the pain their affliction causes without any form of relief their government could provide.
Luckily their ministry has stressed that this was just a rumor and that “it is closely monitoring all medication needs of the sickle cell patients in accordance with the new treatment protocol taken in light of the recommendations of a group of experts from the US-based Johns Hopkins Hospital.”
he press release can be found here: http://www.bna.bh/portal/en/news/704563
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