Merz — Fampyra EMA EPAR public assessment report (EMEA/H/C/002097, 2011)

Report summary

The EMA's CHMP initially rejected Fampyra (fampridine) for improving walking in adult MS patients due to concerns about clinical relevance of efficacy and safety. After re-examination, conditional marketing authorization was granted in May 2011. Non-clinical studies showed acceptable safety, with CNS and urogenital toxicity at high doses. Clinical pharmacokinetic studies highlighted fampridine's narrow therapeutic index and increased exposure in renally impaired and elderly patients. Pivotal Phase 3 studies (MS-F203, MS-F204) demonstrated statistically significant improvements in walking speed (37.3% responder rate vs. 8.9% for placebo). While the clinical relevance of the T25FW test was debated, a 20% improvement was deemed clinically meaningful, correlating with patient-reported outcomes. Safety concerns included increased adverse events, particularly nervous system disorders, seizures (rare but significant), infections, and cardiovascular issues. However, the CHMP concluded the seizure risk was low at therapeutic doses and other adverse events were mild. Long-term safety and efficacy data were deemed sufficient with proposed monitoring. The overall benefit-risk was considered favorable for adult MS patients with EDSS 4-7, addressing an unmet medical need.

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