Zevra Therapeutics (ZVRA) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Strategic vision and pipeline overview

• Focused on rare diseases, with a commercial-stage product (OLPRUVA) and a robust pipeline including arimoclomol (pending approval), KP1077 for idiopathic hypersomnia, and celiprolol for vascular Ehlers-Danlos syndrome.

• Vision includes expanding product reach and addressing unmet needs in rare disease markets over the next five to ten years.

Arimoclomol regulatory and launch plans

• PDUFA date for arimoclomol is September 21, with communication plans ready for early, on-time, or weekend approval.

• Ongoing labeling discussions with the agency, aiming for a label that reflects clinical study data.

• Sufficient product is available for launch, with drug expected in the channel 8–12 weeks post-approval.

• Plan to transition 70 U.S. expanded access patients to commercial product within 12 months of approval.

• Pricing will be based on final label and value demonstrated in clinical trials, aiming to reflect added benefit over existing care.

Market opportunity and patient access

• U.S. prevalence for Niemann-Pick C is about 900, with 300–350 actively treated; 70 are in the expanded access program.

• Goal to expand reach to all eligible patients and educate physicians to improve diagnosis and treatment rates.

• European opportunity to be revisited post-U.S. approval, leveraging a robust regulatory package.

• Orphan exclusivity expected for up to seven years in the U.S. and Europe.