Onfi is a 1,5-benzodiazepine that acts by potentiating gamma-aminobutyric acid (GABA)ergic
transmission through binding at the GABA[A] receptor. However, the precise mechanism of action by
which clobazam exerts its anti-epileptic effects is unknown. It was proposed as an effective
anticonvulsant with an improved side-effect profile compared with 1,4-benzodiazepines, such as
diazepam and clonazepam.
Originally developed by Aventis (now Sanofi), Ovation Pharmaceuticals acquired the developmental
rights to the product in 2004. However, Lundbeck continued the development and commercialization
of the product following its acquisition of Ovation Pharmaceuticals in 2009. The US Food and Drug
Administration’s (FDA’s) approval for Onfi in October 2011 for the treatment of LGS was based on two
multicenter controlled studies in patients aged two years and older (Lundbeck, 2011). Onfi had
previously been designated orphan drug status, and as a result was granted a seven-year exclusivity
period, during which the FDA is prohibited from approving any other formulation for LGS unless the
other formulation is demonstrated to be clinically superior to Onfi.
Prior to Lundbeck’s development of Onfi for LGS, clobazam had already been independently marketed
by Sanofi in Europe as an anticonvulsant since 1984 and as an anxiolytic since 1975. It is marketed
under the name Frisium in most of the EU and as Urbanyl in France, where it is approved for the
short-term relief (2−4 weeks) of severe and disabling anxiety, and as an adjunctive treatment for
epilepsy (Doctissimo, 2016; eMC, 2015; Pharmaprojects, 2016). Furthermore, under license from
Sanofi, Dainippon Sumitomo has also launched clobazam for the treatment of epilepsy under the
brand name Mystan in Japan (Biomedtracker, 2016; Ng and Collins, 2007; Sugai, 2004).
Onfi can cause abuse and dependence and has been categorized as a schedule IV drug under the
Controlled Substances Act. The FDA requires that patients and caregivers receive a medication guide
describing the risks and adverse reactions associated with the drug (FDA, 2011).
Despite its niche indication, Onfi’s (clobazam; Lundbeck) sales in the US are experiencing growth
following its approval in 2011. Despite being approved after Banzel (rufinamide; Eisai) for a similar
niche indication, limited to Lennox-Gastaut syndrome (LGS), Onfi is significantly outperforming Banzel
in US sales. This is largely driven by the long history of clinical experience with clobazam outside of
the US and Onfi’s strong efficacy data that have contributed to its popularity and commercial success
TABLE OF CONTENTS
4 PRODUCT PROFILES
4 Onfi : Epilepsy
LIST OF FIGURES
9 Figure 1: Onfi for epilepsy – SWOT analysis
11 Figure 2: Datamonitor Healthcare’s drug assessment summary for Onfi in epilepsy
11 Figure 3: Datamonitor Healthcare’s drug assessment summary for Onfi in epilepsy
LIST OF TABLES
4 Table 1: Onfi drug profile
7 Table 2: Onfi pivotal trial data in epilepsy
9 Table 3: Safety profile for Onfi in epilepsy
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