Rare Disease Report

Top 5 Rare Disease News of the Week—June 24, 2018

JUNE 30, 2018
Rare Disease Report® Editorial Staff

#5: Phase 3 Trial Evaluating Voxelotor for Sickle Cell Disease Boasts Positive Top-Line Data

Positive top-line data from Part A of Global Blood Therapeutics, Inc.’s phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) trial evaluating voxelotor for the treatment of sickle cell disease (SCD) were released.

Results showed a statistically significant increase in hemoglobin in those who received voxelotor in either 1500 mg or 900 mg doses after 12 weeks of treatment compared with placebo.

Read more about the sickle cell disease treatment.  

#4: Updated Data Released Regarding Use of Edasalonexent in DMD Patients

Released data from the MoveDMD phase 2, open-label, extension trial assessing edasalonexent in boys diagnosed with Duchenne muscular dystrophy (DMD) were announced.

The investigators noted a significant decrease in CRP with edasalonexent treatment, which further supports the biological activity of NF-kB inhibition by treatment with the drug decreasing inflammation. Furthermore, compared with the rate of change in the off-treatment control period, edasalonexent substantially slowed DMD disease progression in boys on 100 mg/kg through more than a year of treatment.

Read more about the MoveDMD trial.  

#3: FDA Grants Priority Review to Waldenstrom's Macroglobulinemia Combination Therapy

The US Food and Drug Administration (FDA) granted Priority Review for a supplemental New Drug Application (sNDA) for the Waldenström's macroglobulinemia combination therapy, ibrutinib (IMBRUVICA) in combination with rituximab (RITUXAN).

Read more about the new Waldenstrom's macroglobulinemia treatment.

#2: FDA Approves Cannabidiol for Lennox-Gastaut Syndrome and Dravet Syndrome

The FDA approved cannabidiol (Epidiolex) [CBD] oral solution in patients aged 2 years and older for the treatment of severe and rare types of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome.

CBD, more commonly known as marijuana, is a chemical component of the cannabis sativa plant. However, intoxication or euphoria (the “high”) that comes from tetrahydrocannabinol (THC), which is the primary psychoactive component of marijuana. This was the first-approved marijuana-derived medication.

Read more about the newly approved Lennox-Gastaut Syndrome and Dravet Syndrome treatment.

#1: Dr Takami Sato Discusses IMCgp100 Treatment for Metastatic Uveal Melanoma

In a recent interview with Rare Disease Report®, Takami Sato, MD, PhD, from the department of Medical Oncology at Kimmel Cancer Center, Thomas Jefferson University, discussed the use of IMCgp100 as a treatment for metastatic uveal melanoma (mUM).

IMCgp100 is a bispecific biologic comprised of a soluble T-cell receptor recognizing the gp100 antigen fused to a scFV anti-CD3 that redirects T-cell lysis of melanoma cells expressing gp100, in mUM. The estimated primary completion date for its phase 1/2 study is September 2019.

Read more about the metastatic uveal melanoma (mUM) treatment.

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