UCB Receives Positive Opinion from European Authorities for Orphan Designation of Brivaracetam for the Treatment of Progressive Myoclonic Epilepsies

Brussels (Belgium) July 25, 2005: UCB announced today that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA), has adopted a positive opinion recommending the granting of orphan medicinal product designation for brivaracetam in the treatment of progressive myoclonic epilepsies. "This positive opinion represents an important step in UCB's development of brivaracetam, and demonstrates our commitment to the development of significant treatment options for patients with difficult-to-treat epilepsies." said Roch Doliveux, CEO of UCB. 

Progressive myoclonic epilepsies (PMEs) are a group of symptomatic generalised epilepsies caused by rare disorders, most of which have a genetic component, a debilitating course and a poor outcome. Challenges with PME arise from difficulty with diagnosis and problems of management[1].

Brivaracetam is a SV2A ligand that has shown significant antiepileptic activity in animal models of epilepsy, both in vitro and in vivo[2,3] as well as in a photosensitive epilepsy model in humans[4]. Brivaracetam is currently being evaluated for the treatment of refractory patients with partial onset seizures.

Orphan medicinal products are used to diagnose, prevent or treat life-threatening or very serious conditions that are rare, with a prevalence of less than five per 10,000 of the EU population. The EMEA, through the COMP is responsible for reviewing designation applications from sponsors who intend to develop medicines for rare diseases. European orphan drug designation enables recipient sponsors to receive regulatory guidance in the drug development process and allows for up to 10 years of European market exclusivity for the designated indication upon approval of the market application.

REFERENCES

1. Shahwan, A., Farrell, M., Delanty, N. Progressive Myoclonic Epilepsies: a review of genetic and therapeutic aspects Lancet Neurol 2005; 4(4): 239-48
2. Lynch, B.A., Lambeng, N., Nocka, K. et al. The synaptic vesicle protein SV2A is the binding site for the antiepileptic drug levetiracetam PNAS 2004; 101(26): 9861-9866
3. Kenda, B.M., Matagne, A.C., Talaga, P.E. et al. Discovery of 4-substituted pyrrolidone butanamides as new agents with significant antiepileptic activity J. Med. Chem. 2004; 47(3): 539-549
4. Kasteleijn-Nolst Trenite DGA, Parain D, Masnou P et al. Proof of principle in the new AED, UCB 34714; use of the photosensitivity model. Presentation at the 58th American Epilepsy Society Congress, New Orleans, 7th December 2004