Experimental sickle cell disease drug gets PRIME status

Global Blood Therapeutics’ GBT440 has become the first potential treatment for sickle cell disease to be accepted onto the European Medicines Agency’s regulatory support programme PRIME.

SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of haemoglobin, which leads to the formation of abnormal haemoglobin which binds together to form long, rigid rods within a red blood cell.

Patients with the condition suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities.

GBT440 is being developed as an oral, once-daily therapy for SCD. The drug works by increasing haemoglobin's affinity for oxygen, which the firm believes will prevent ‘sickling’ of red blood cells and potentially modify the course of the disease.

The PRIME programme provides early and proactive EMA support to medicine developers, the idea being to speed up development and thus help patients benefit as early as possible from innovative new products that have demonstrated the potential to significantly address an unmet medical need.

"Access to the EMA’s PRIME programme is important not just for GBT, but for the entire SCD community, as this is the first eligibility granted to PRIME for a SCD therapy. It provides further recognition from a regulatory authority that SCD is a severely unmet medical need that requires attention," said Ted Love, president and chief executive officer of GBT.

"Additionally, it is external validation of the potential of GBT440 to address this need and bring a major therapeutic advantage to patients," he noted.