FDA Grants GlycoMimetics Rare Pediatric Disease Designation for Rivipansel for Treatment of Sickle Cell Disease
October 05 2020 - 4:30PM
Business Wire
GlycoMimetics, Inc. (Nasdaq: GLYC) today announced that the U.S.
Food and Drug Administration (FDA) has granted the Company a Rare
Pediatric Disease designation for rivipansel for the treatment of
sickle cell disease in patients 18 years old and younger. This
designation recognizes the significant needs in pediatric
patients.
“The FDA’s designation recognizes the morbidity and mortality
burdens of sickle cell disease as well as its significant impact
during childhood with life-long implications. With this
designation, the agency acknowledges the urgent need for improved
treatment of children living with sickle cell disease,” stated
Helen Thackray, Chief Medical Officer of GlycoMimetics.
“With global rights for rivipansel in our hands, we are
exploring options to move forward in sickle cell disease, including
discussions with the FDA as to whether there is a regulatory path
to approval,” added Rachel King, Chief Executive Officer. “We plan
to roll out the full data set from the Phase 3 RESET program at
upcoming medical meetings, based on the acceptance of abstract
submissions.”
About Rare Pediatric Disease Designation
The FDA defines a "rare pediatric disease" as a serious or
life-threatening rare disease in which the serious or
life-threatening manifestations primarily affect individuals aged
from birth to 18 years. Under the FDA's Rare Pediatric Disease
Priority Review Voucher program, a sponsor who receives an initial
approval for a drug or biologic for a “rare pediatric disease” may
qualify for a voucher that can be redeemed to receive a priority
review of a subsequent marketing application for a different
product.
About Sickle Cell Disease (SCD) and VOC
SCD is the most common inherited blood disorder in the United
States, impacting approximately 100,000 people. Worldwide,
approximately 100 million people carry the SCD trait and an
estimated five million live with the disease. While the majority of
people with SCD are of African descent, the disease can affect all
ethnic groups, especially those from areas where malaria is or was
endemic, such as the Middle East, India and the Southern
Mediterranean. Acute pain crises or VOCs are the most common
clinical manifestation of SCD. A VOC occurs when hypoxia and
inflammation lead to vascular occlusion, tissue ischemia and
pain.
About Rivipansel
Rivipansel, a glycomimetic drug candidate that binds to all
three members of the selectin family (E-, P- and L-selectin), was
GlycoMimetics’ first drug candidate to enter clinical development.
After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’
former collaborator, produced disappointing results in 2019, new
clinical outcome data from a post hoc analysis of early treatment
with rivipansel were published in June 2020 in advance of a poster
shown at the Foundation for Sickle Cell Disease Research Meeting on
September 24, 2020.
About GlycoMimetics, Inc.
GlycoMimetics is a biotechnology company with two late-stage
clinical development programs and a pipeline of novel glycomimetic
drugs, all designed to address unmet medical needs resulting from
diseases in which carbohydrate biology plays a key role.
GlycoMimetics' drug candidate, uproleselan, an E-selectin
antagonist, was evaluated in a Phase 1/2 clinical trial as a
potential treatment for acute myeloid leukemia (AML) and is being
evaluated across a range of patient populations including a
Company-sponsored Phase 3 trial in relapsed/refractory AML under
breakthrough therapy designation. Rivipansel, a pan-selectin
antagonist, is being explored for use in treatment of acute VOC in
SCD. GlycoMimetics has also completed a Phase 1 clinical trial with
another wholly-owned drug candidate, GMI-1359, a combined CXCR4 and
E-selectin antagonist. GlycoMimetics is located in Rockville, MD in
the BioHealth Capital Region. Learn more at
www.glycomimetics.com.
Forward-Looking Statements
This press release contains forward-looking statements regarding
the clinical development and potential benefits and impact of the
Company’s drug candidates. These forward-looking statements include
those relating to the planned or potential clinical development of
the Company’s product candidates, including the presentation of
data from clinical trials. Actual results may differ materially
from those described in these forward-looking statements. For a
further description of the risks associated with these statements,
as well as other risks facing GlycoMimetics, please see the risk
factors described in the Company’s annual report on Form 10-K filed
with the U.S. Securities and Exchange Commission (SEC) on February
28, 2020, and other filings GlycoMimetics makes with the SEC from
time to time. Forward-looking statements speak only as of the date
of this release, and GlycoMimetics undertakes no obligation to
update or revise these statements, except as may be required by
law.
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