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Zavegepant Intranasal

Phase 3 Pivotal Clinical Trial of Zavegepant, a Calcitonin Gene-Related Peptide (CGRP) Receptor Antagonist Nasal Spray for the Acute Treatment of Migraine

Year of Publication: 2023

Authors: Richard B. Lipton, M.D. (Lead Author), et al.

Journal: The Lancet Neurology

Citation: Published February 16, 2023 in The Lancet Neurology

Link: https://www.businesswire.com/news/home/20230216005209/en/


Clinical Question

Is zavegepant 10 mg intranasal spray effective and safe for the acute treatment of migraine in adults compared to placebo?

Bottom Line

Zavegepant 10 mg intranasal spray demonstrated superiority over placebo for acute treatment of migraine, achieving significantly higher rates of pain freedom and freedom from most bothersome symptom at 2 hours post-dose, with pain relief beginning as early as 15 minutes and lasting up to 48 hours. The treatment was well tolerated with no serious adverse events.

Major Points

  • First Phase 3 trial of a non-oral CGRP receptor antagonist for acute migraine treatment
  • Met both co-primary endpoints: pain freedom at 2 hours (24% vs 15%, P<0.0001) and freedom from most bothersome symptom at 2 hours (40% vs 31%, P=0.0012)
  • Demonstrated rapid onset of pain relief at 15 minutes with sustained benefits through 48 hours
  • Well tolerated with dysgeusia (altered taste) being the most common adverse event (20.5% vs 4.7% placebo)
  • No serious adverse events or hepatotoxicity signals identified
  • Zavegepant is a third generation, high affinity, selective small molecule CGRP receptor antagonist

Design

Study Type: Randomized, placebo-controlled, double-blind trial

Randomization: 1

Blinding: Double-blind

Follow-up Duration: 48 hours post-dose

Centers: 0

Countries: United States

Sample Size: 1405

Analysis: Superiority analysis with hierarchical testing of secondary endpoints


Inclusion Criteria

  • Adults with migraine
  • History of 2-8 moderate or severe migraine attacks per month
  • Untreated attacks lasting a mean of 30.8 hours

Arms

FieldControlZavegepant 10 mg
InterventionPlacebo intranasal sprayZavegepant 10 mg intranasal spray
DurationSingle doseSingle dose

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Co-primary endpoints: Pain freedom at 2 hours post-dose and freedom from most bothersome symptom at 2 hours post-dosePrimary15% pain freedom, 31% freedom from MBS24% pain freedom, 40% freedom from MBS9.00%P<0.0001 for pain freedom; P=0.0012 for freedom from MBS
Pain relief at 15 minutesSecondaryStatistically significant
Sustained pain relief from 2 through 48 hours post-doseSecondaryStatistically significant
Return to normal function at 15 minutes post-doseSecondaryNot statistically significantNS
Return to normal functional ability at 30 minutesSecondaryHigher rates with zavegepant
Return to normal functional ability at 2 hoursSecondaryHigher rates with zavegepant
13 of 17 secondary endpoints showed superiority to placeboSecondary
20.5% zavegepant vs 4.7% placeboAdverse
3.7% zavegepant vs 0.8% placeboAdverse
3.2% zavegepant vs 1.1% placeboAdverse
None in either groupAdverse
No signal identifiedAdverse

Criticisms

  • One secondary endpoint (return to normal function at 15 minutes) did not reach significance, preventing formal testing of remaining endpoints per hierarchical analysis plan
  • Exclusion criteria not detailed in the press release
  • Limited follow-up duration for long-term safety assessment
  • No comparison to active comparators (e.g., triptans)
  • Baseline characteristics not fully detailed by treatment arm

Funding

Pfizer Inc.

Based on: Zavegepant Intranasal (The Lancet Neurology, 2023)

Authors: Richard B. Lipton, M.D. (Lead Author), et al.

Citation: Published February 16, 2023 in The Lancet Neurology

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