Rare Disease Report


Positive developmental updates on the investigational BTK inhibitor, zanubrutinib, as a treatment for Waldenström macroglobulinemia were recently released.
The FDA has granted orphan drug designation to Oncology Venture AB for their oral phase 3 multi tyrosine kinase inhibitor (previously TKI258), dovitinib DRP, for the treatment of adenoid cystic carcinoma (AdCC).
The US Food and Drug Administration (FDA) has approved encorafenib and binimetinib (BRAFTOVI and MEKTOVI) in combination for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation as detected by an FDA-approved test.
Takami Sato, MD, PhD, Department of Medical Oncology, Kimmel Cancer Center, Thomas Jefferson University, discusses new treatment for metastatic uveal melanoma: IMCgp100.
The 2018 American Society of Clinical Oncology (ASCO) Annual Meeting held in Chicago, Illinois, June 1-5, 2018, featured data from numerous studies and advancements being made in rare cancers. Here are 5 key takeaways from the meeting.  
hATTR compass program will empower people with accurate genetic information so they can make informed decisions about their healthcare.
Nicholas J Robert, MD, discusses treatment challenges associated with rare cancers like Merkel cell carcinoma.
Updated interim data from Affimed NV’s phase 1b dose-escalation study evaluating AFM13 for the treatment of relapsed/refractory Hodgkin Lymphoma were released at the 23rd Congress of the European Hematology Association.
Ray Comenzo, MD, highlights promising ANDROMEDA phase 3 trial, which evaluated the efficacy of subcutaneous daratumumab and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy for newly diagnosed light chain (AL) amyloidosis patients.
William D. Tap, MD, lead investigator of the ENLIVEN study provides latest update on pexidartinib as an effective treatment for patients with tenosynovial giant cell tumor (TGCT), especially the diffuse form.
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