Rare Disease Report

Top 5 Rare Disease News of the Week—July 1, 2018

JULY 07, 2018
Rare Disease Report® Editorial Staff


#5: FDA Grants Fast Track Designation to CUTX-101 for Classic Menkes Disease

The US Food and Drug Administration (FDA) granted Cyprium Therapeutics, a subsidiary of Fortress Biotech, Inc, Fast Track Designation for its product, CUTX-101, for the treatment of patients with classic Menkes disease who have not demonstrated significant clinical progression. 

CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP, according to a recent news release. It is intended to improve tolerability due to physiological pH as well as bypass the oral absorption of copper, a functionality that is impaired in patients with Menkes disease.

Read more about the developing Menkes disease treatment.

#4: Update on Waldenström Macroglobulinemia Treatment Reports Positive Findings

Updated data from the phase 1 trial of zanubrutinib as a monotherapy in patients with different subtypes of B-cell malignancies and a pooled analysis of the safety data from the zanubrutinib monotherapy trials were released.

Zanubrutinib was found to be effective with an overall response rate of 92% and a major response rate of 80%. Additionally, the 12-month progression-free survival rate was estimated to be 91%.  

Read more about the positive findings from the zanubrutinib updates.  

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