Rare Disease Report

Oncology

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.
This morning, Novartis was granted U.S. FDA Priority Review for the drug in patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for or relapse after autologous stem cell transplant (ASCT).
Ipsen and Exelixis, Inc. announced detailed results of the pivotal phase 3 CELESTIAL trial, evaluating cabozatinib in previously treated patients with advanced hepatocellular carcinoma.
In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.
Boehringer Ingelheim announced the approval of a new indication for afatinib, as the U.S. FDA approved a supplemental NDA for the first-line treatment of patients with metastatic NSCLC whose tumors have non-resistant EGFR mutations.
The U.S. Food and Drug Administration has approved a third indication for olaparib tablets (Lynparza).
In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.
At the 36th Annual J.P. Morgan Healthcare Conference, uniQure announced that in 2018, the company intends to advance its gene therapy AMT-061 into a pivotal study for hemophilia B.
BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.
Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.
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