Rare Disease Report


The FDA has granted a rare pediatric disease designation to CLR 131 for the treatment of Ewing’s sarcoma. 
Investigators find boys treated with alkylating agents or with hydroxyurea for sickle cell disease resulted in a significant reduction in spermatogonial cell counts, suggesting that these agents can affect fertility.
The FDA has approved mogamulizumab-kpkc (Poteligeo) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least 1 prior systemic therapy. 
The US Food and Drug Administration (FDA) has granted an orphan drug designation to Alteogen Inc.’s ALT-P7 for the treatment of patients with gastric cancer.
The FDA has granted a fast track designation to Nohla Therapeutics’ dilanubicel (NLA101) for patients with high-risk hematologic malignancies receiving an allogeneic cord blood transplant.
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
The FDA has granted a breakthrough therapy designation to Daiichi Sankyo Company, Limited’s investigational FLT3 inhibitor, quizartinib, for the treatment of adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML).
Researchers investigate whether the location of a mutation in the PIK3CA gene affects the mutation’s ability to drive cancerous growth.
Manmeet Ahluwalia, MD, discusses the challenges patients and providers face when it comes to rare cancers.
The FDA granted an orphan designation to Surface Oncology’s SRF231 for the treatment of multiple myeloma, a rare blood cancer.
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