Recent Treatment Advances Made in the Fight Against Sickle Cell Disease

In honor of World Sickle Cell Awareness Day, we’ve rounded up the big treatment advances made this year thus far for sickle cell disease (SCD), a disease that affects over 300,000 babies each year.

Sickle cell disease is a group of inherited red blood cell disorders, according to the Centers for Disease Control and Prevention. Those with the disease have red blood cells that are hard, sticky, and C-shaped; the misshapen cells clog smaller blood vessels, resulting in excruciating pain.

On this international awareness day, held each year on June 19, researchers spanning all specialties and patients from all backgrounds come together to share experiences and increase public knowledge and understanding of the disease.

Here’s our round-up of advances made thus far in 2018 for the treatment of SCD:

Sickle Cell Treatment Becomes Available in the United States

At the beginning of 2018, Emmaus Life Sciences announced that Endari, a medication developed to decrease acute complications in patients with SCD was made available by prescription in the United States.

Endari had been approved by the US Food and Drug Administration (FDA) back in July 2017 for pediatric patients aged 5 and older with SCD. Previous to this approval, hydroxyurea had been the main treatment available for suppressing pain episodes. Endari is known to be the first FDA-approved treatment for adult patients in 20 years and the first treatment available ever for pediatric patients.

Read more about Endari.

First Sickle Cell Patient Dosed in Phase 2a Trial of IMR-687

In February 2018, Imara Inc. announced the dosing of their first patient in their phase 2a clinical trial designed to evaluate the safety, pharmacokinetics and pharmacodynamics of escalating doses of IMR-687 in adult patients with SCD.

The drug was granted Rare Pediatric Disease Designation by the FDA in May 2017, making it the first drug candidate for SCD to receive the label. Investigators are developing the drug to be used as a highly-potent oral therapy to be dosed once-daily to address the underlying red and white blood cell pathologies associated with the condition.

Read more about the phase 2a trial of IMR-687.

European Commission Grants Orphan Drug Designation to Altemia for Treatment of SCD

In April 2018, the European Commission granted Sancilio Pharmaceuticals Company, Inc. orphan drug designation for Altemia, a novel oral formulation being developed for the treatment of SCD in pediatric patients. The FDA also granted orphan drug designation to the drug back in 2015.

Altemia is a mixture of lipids encased in a small, soft gelatin capsule intended to be taken once-daily to reduce anemia, organ damage, vaso-occlusive crises (VOC) episodes, and other associated complications. The drug was developed to replace the specific lipids HbS destroys in an effort to prevent the initiation of the cascade effect that occurs in VOC episodes.

Read more about Altemia.

FDA Accepts IND Application for Gene Therapy to Treat SCD

In May 2018, Bioverativ, Inc. announced that the FDA accepted the company’s Investigational New Drug (IND) application for their gene-edited cell therapy candidate, BIVV003, for the treatment of those with SCD.

The acceptance marked the second IND for a gene-editing approach in less than a year, as well as the first ever for a gene-edited therapy intended to fight SCD. Biovertaiv and Sangamo are working to progress and commercialize these therapies for SCD and beta thalassemia.

Read more about the gene therapy for sickle cell disease.