Rare Disease Report


The U.S. Food and Drug Administration (FDA) has granted accelerated approval to blinatumomab (Blincyto) for the treatment of adults and children with B-cell precursor ALL who are in remission, but still have minimal residual disease.
A new study concludes that, among patients with AML, the detection of molecular minimal residual disease during complete remission had significant independent prognostic value with respect to relapse and survival rates.
Results from a trial sponsored by the National Cancer Institute demonstrate that sorafenib tosylate (Nexavar) extended progression-free survival in patients with desmoid tumors or aggressive fibromatosis.
The U.S. FDA announced that Breakthrough Therapy Designation had been granted to Fennec Pharmaceuticals for its unique formulation of cisplatin and sodium thiosulfate (STS).
Data from a Phase 2 study suggests that voxtalisib has a promising safety profile, but might not be as effective in patients with aggressive malignancies as it is in patients with follicular lymphoma.
The U.S. FDA has expanded the approval for nilotinib to include the treatment of first- and second-line pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP).
The U.S. FDA has granted Pedmark, a unique combination of cisplatin and sodium thiosulfate, Fast Track designation for the prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma.
The U.S. FDA has approved brentuximab vedotin (Adcetris) in combination with chemotherapy for adult patients with previously untreated Stage III or IV classical Hodgkin lymphoma.
This morning, Cellectar Biosciences announced that the U.S. FDA has granted orphan drug designation to CLR 131 for the treatment of neuroblastoma.
Phase 2 clinical and immunological data presented at the 44th Annual EBMT Meeting this morning revealed that galinpepimut-S is safe and effective in high-risk multiple myeloma.
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