SICKLE CELL DISEASE (SCD) AND VOC

New Pediatric and Secondary Endpoint Data from Rivipansel Phase 3 RESET Trial Presented at The Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) Meeting

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Monday, October 26, 2020

Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical trials, Sickle Cell Disease (SCD) and VOC, Rivipansel, GMI-1687, GlycoMimetics, Inc.

The favorable safety profile of rivipansel observed in the Phase 3 RESET trial, as evaluated in a population with pediatric, adolescent, and adult patients, is highly encouraging to us.

Key Points:

The favorable safety profile of rivipansel observed in the Phase 3 RESET trial, as evaluated in a population with pediatric, adolescent, and adult patients, is highly encouraging to us.
In addition, newly scheduled for presentation at this meeting are data for the pediatric subgroup, who make up a large proportion (41%) of patients treated in the RESET trial.
The abstract also features key findings previously reported at the Foundation for Sickle Cell Research (FSCDR) meeting in September, 2020.
Data from the RESET trial additionally demonstrate a safety profile for rivipansel that is comparable to the placebo.

FDA Grants GlycoMimetics Rare Pediatric Disease Designation for Rivipansel for Treatment of Sickle Cell Disease

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Monday, October 5, 2020

Health, FDA, Consumer, Teens, Children, Pharmaceutical, Biotechnology, Medicine, Articles, Branches of biology, Health in Africa, Hematopathology, RTT, Sickle cell disease, Disease, Rare Pediatric Disease Designation, Sickle Cell Disease (SCD) and VOC, Rivipansel, GlycoMimetics, Inc.

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.

Key Points:

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.
The FDAs 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.
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.

New Efficacy and Biomarker Data From Rivipansel Phase 3 RESET Trial to Be Presented at Sickle Cell Meeting

Retrieved on:

Friday, June 12, 2020

This analysis and new biomarker data will be presented at the September meeting of the Foundation for Sickle Cell Disease Research (FSCDR).

Key Points:

This analysis and new biomarker data will be presented at the September meeting of the Foundation for Sickle Cell Disease Research (FSCDR).
Furthermore, patients treated with rivipansel showed a statistically significant reduction in soluble E-selectin, a biomarker indicating that the drug had the intended biological effect.
Data from the RESET trial additionally demonstrate a safety profile for rivipansel comparable to the placebo.
In addition, the biomarker data showing reductions in soluble E-selectin indicates that rivipansel is hitting its intended biological target.