Rare Disease Report
Patients & Caregivers


The FDA has accepted Genentech’s supplemental Biologics License Application and granted Priority Review for follicular lymphoma treatment Gazyva (obinutuzumab).
Breakthrough Therapy Designation status was granted to mogamulizumab to treat subtytes of cutaneous T-cell lymphoma – mycosis fungoisis and Sézary syndrome.
Bucket list items: 1) survive cancer; 2) meet Tom Brady; 3) attend Patriots game 

In February 2016, the FDA approved Afinitor (everolimus) for treating neuroendocrine tumors (NETs) of gastrointestinal or lung origin.
The verdict included $70 million in compensatory damages and $347 million in punitive damages.
Syros announced that the U.S. FDA has granted orphan drug designation to its leading drug candidate SY-1425, intended to treat acute myeloid leukemia (AML).
A Phase 1 clinical study testing CAR T-cell therapy, UCART123,to treat blastic plasmacytoid dendritic cell neoplasm at the MD Anderson Cancer Center has begun.
Curtana announced the FDA has granted CT-179 with an Orphan Drug Designation to treat for gliomas, including glioblastoma in adults and brain tumors in children.
The FDA approved olaparib tablets (Lynparza) for adult patients with recurrent epithelial ovarian fallopian tube, or primary peritoneal cancer.
FDA has approved Besponsa (inotuzumab ozogamicin) for treating adults with relapsed or refractory B-cell precursor ALL
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