Sentences with phrase «certain gene therapy products»

Among other things, we plan to include certain gene therapy products that permanently alter tissue and produce a sustained therapeutic benefit as part of the products that will meet the definition of being eligible to come under the pathway enabled by RMAT.

Not exact matches

Endari, the first new treatment for patients with sickle cell disease in almost 20 years, Genentech's Hemlibra, the first - ever non-blood product to treat patients with hemophilia A with inhibitors, Actemra, the first treatment for adults diagnosed with giant cell arteritis, BioMarin's Brineura, the first treatment for a form of Batten disease, Benznidazole, the first U.S. treatment for Chagas disease, Novartis» Kymriah to treat certain children and young adults with B - cell acute lymphoblastic leukemia, which is also the first gene therapy to become available in the United States, are some of the drugs that received the FDA's stamp of approval in 2017.