- Clinical Trials
- Research News
- Industry Trends
- Agency Actions
- Drug Safety Issues
- Approvals, Launches, & New Indications
- Health Care Reform
FDA Panel Questions Safety of MS Drug Alemtuzumab (Lemtrada)
Announcement casts doubt on approval decision (November 8)
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has raised concerns about “multiple serious and potentially fatal safety issues” in patients treated with the new multiple sclerosis (MS) drug alemtuzumab (Lemtrada, Genzyme Corp.), casting doubt on the drug’s upcoming approval decision.
Alemtuzumab’s proposed indication is for the treatment of patients with relapsing forms of MS to slow or reverse the accumulation of physical disability and to reduce the frequency of clinical exacerbations.
The announcement was made prior to a formal advisory board meeting scheduled for November 13. At the meeting, experts will discuss safety concerns associated with the use of alemtuzumab for the treatment of MS. These concerns include the incidence of an array of autoimmune diseases, including immune thrombocytopenia (ITP), autoimmune hemolytic anemia, immune pancytopenia, anti-glomerular basement membrane (anti-GBM) disease, membranous glomerulonephritis, thyroid disorders, endocrine ophthalmopathy, acquired hemophilia A, type 1 diabetes mellitus, acute epitheliopathy of the retina, autoimmune skin disease, and undifferentiated connective-tissue disorders, along with the incidence of malignancies, notably thyroid cancer and melanoma.
In addition, panel members will review various concerns associated with the data presented by Genzyme in support of a demonstration of clinical benefit. These concerns stem from issues involved with the adequacy of the design of the primary trials on which the application relies for support. In particular, panel members have concerns that the failure to blind patients and treating physicians in the open-label design of the trials introduced bias that confounds interpretation of their apparent results.
Source: FDA; November 8, 2013.