Several agents receive orphan drug designation

Issue: June 10, 2017

The FDA granted orphan drug designation to several agents designed to treat hematologic or oncologic conditions.

These include:

BI 836858 (Boehringer Ingelheim), an anti-CD33 monoclonal antibody in development for the treatment of myelodysplastic syndrome;
eFT508 (eFFECTOR Therapeutics), a highly selective inhibitor of MNK1 and MNK2 in development for treatment of diffuse large B-cell lymphoma;
Ganitumab (AMG 479, NantCell), a fully human monoclonal antibody directed against insulin growth factor-1R in development for treatment of Ewing sarcoma;
Larotrectinib (LOXO-101, Loxo Oncology), intended to treat solid tumors with neurotrophic tyrosine receptor kinase–fusion proteins;
SB-525 cDNA (Sangamo Therapeutics) gene therapy, which uses an adeno-associated virus that carries a clotting Factor VIII gene construct. The therapy is intended for the treatment of hemophilia A; and
Yeliva (ABC294640, RedHill Biopharma), a first-in-class, orally administered, sphingosine kinase-2 selective inhibitor with anticancer and anti-inflammatory activities in development for the treatment of cholangiocarcinoma.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

For more information about these agents or other regulatory actions, visit Healio.com/HemOnc and search “FDA news.