Erenumab for Migraine Prevention in a 1-Year Compassionate Use Program: Efficacy, Tolerability, and Differences...

Area of Science:

• Neurology
• Pharmacology
• Clinical Trials

Background:

• Migraine is a debilitating neurological disorder affecting millions worldwide.
• Existing prophylactic treatments have limitations, necessitating novel therapeutic approaches.
• Erenumab targets the calcitonin gene-related peptide (CGRP) pathway, a key mediator in migraine pathophysiology.

Purpose of the Study:

• To evaluate the real-world effectiveness and safety of monthly erenumab 140 mg in patients with episodic migraine (EM) and chronic migraine (CM).
• To assess treatment response across different migraine severities and prior treatment histories.
• To determine the long-term (12-month) efficacy and tolerability of erenumab.

Main Methods:

• A 1-year compassionate use program involving 156 patients with migraine.
• Patients self-administered monthly subcutaneous erenumab 140 mg.
• Inclusion criteria: ≥ 4 migraine days/month and ≥ two prior prophylactic treatment failures.

Main Results:

• Significant reductions in monthly headache days observed by month 3 (45.7% in EM, 35.5% in CM).
• 50% responder rates at 12 months were 51% for EM and 41% for CM.
• Constipation was the most frequent non-serious adverse event (20.5% at 3 months, 15% at 12 months); discontinuation due to adverse effects was rare (3.8%).

Conclusions:

• Monthly erenumab 140 mg demonstrates high effectiveness for migraine prophylaxis across the disease spectrum, except in patients with continuous headaches.
• Therapeutic effects are rapid, significant after the first month, and sustained over 12 months.
• Erenumab is generally well-tolerated, with constipation being the primary adverse event.