Press Release
Global Blood Therapeutics Receives EMA PRIME Designation for GBT440 for the Treatment of Sickle Cell Disease (SCD)
– GBT440 is First Potential Treatment for SCD To Be Accepted for PRIME –
– PRIME Designation Allows for Frequent and Early Interactions with the Agency Aimed at Assisting Accelerated Evaluation and Approval –
“Access to the EMA’s PRIME program 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
To be accepted for PRIME, a therapy must demonstrate the potential to benefit patients with unmet medical needs through early clinical data. The GBT440 acceptance was supported by data from the ongoing Phase 1/2 clinical trial (GBT440-001) evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of GBT440 in both healthy subjects and adults with SCD. Additionally, results from this trial, coupled with the well understood molecular pathophysiology of SCD and the mechanism of action of GBT440, support the scientific rationale that improvement in hemolysis and hemoglobin may be likely to translate into an improvement in patient symptoms and important disease modification.
About the Priority Medicines (PRIME) Initiative
PRIME is a program launched by the
About Sickle Cell Disease (SCD)
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). In its deoxygenated state, HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (
About GBT440 in Sickle Cell Disease
GBT440 is being developed as an oral, once-daily therapy for patients with SCD. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes GBT440 blocks polymerization and the resultant sickling of red blood cells. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT believes that GBT440 may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (
About
Forward-Looking Statements
Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. We intend these forward-looking statements, including statements regarding the therapeutic potential and safety profile of GBT440, our ability to implement our clinical development plans for GBT440 in both SCD and hypoxemic pulmonary disorders, our ability to generate and report data from our ongoing studies of GBT440, regulatory review and actions relating to GBT440, and the timing of these events, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, the risks that our clinical and preclinical development activities may be delayed or terminated for a variety of reasons, that regulatory authorities may disagree with our clinical development plans or require additional studies or data to support further clinical investigation of our product candidates, and that drug-related adverse events may be observed in later stages of clinical development, along with those risks set forth in our Annual Report on Form 10-K for the fiscal year ended
Myesha Lacy (investors) GBT 650-351-4730 investor@globalbloodtx.comJulie Normart (media)Pure Communications 415-946-1087 media@globalbloodtx.com