Article Content

The U.S. Food and Drug Administration has granted Orphan Drug status to ABT-414 and mocetinostat, used to treat, respectively, glioblastoma multiforme and diffuse B-cell lymphoma (DBCL).

Figure. No caption a... - Click to enlarge in new windowFigure. No caption available.

ABT-414, marketed by AbbVie Oncology, is an investigational compound that is an anti-epidermal growth factor receptor antibody drug conjugate. Glioblastoma multiforme is the most common and most aggressive type of malignant primary brain tumor, with a five-year survival rate for patients in the U.S. and Europe diagnosed with the disease of only about four percent, according to statistics from the National Brain Tumor Society.


Data from a Phase I clinical trial evaluating ABT-414 for the treatment of patients with recurrent or unresectable glioblastoma multiforme were presented this spring at the American Society of Clinical Oncology Annual Meeting (Abstract 2021), and Phase II trials are currently ongoing.


Mocetinostat, made by Mirati Therapeutics, Inc., is a spectrum-selective histone deacetylase (HDAC) inhibitor that reverses aberrant acetylation resulting from HDAC mutations and is predicted to halt tumor progression and reduce tumor burden in patients. Phase II studies for mocetinostat as a single agent are currently being planned for the treatment of patients with HAT mutations in DLBCL, with initial data expected by the end of the year, the company noted in a news release.


Mocetinostat was also granted Orphan Drug designation in June for the treatment of patients with myelodysplastic syndromes (MDS), and is currently in Phase II clinical studies to be used in combination with azacitidine (Vidaza) for patients with intermediate- and high-risk MDS.


Orphan drug designation is also being sought for the treatment of patients with bladder cancer with HAT mutations, thought to be involved in the pathogenesis and progression of such tumor types, according to information from the company.


The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.