Drug Profile

MYR 101

Alternative Names: AVASPA; MYR-101; rAAV Olig001 ASPA - Myrtelle; rAAV-Olig001-ASPA

Latest Information Update: 16 Jul 2025

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At a glance

Originator Pfizer
Developer Myrtelle
Class Gene therapies
Mechanism of Action Amidohydrolase replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Canavan disease
New Molecular Entity No

Highest Development Phases

Phase I/II Canavan disease

Most Recent Events

08 Jul 2025 Myrtelle announces intention to launch MYR 101 for Canavan disease (Intracerebral)
08 Jul 2025 Myrtelle announces intention to submit regulatory filings for MYR 101 for Canavan disease (Intracerebral)
02 Apr 2024 rAAV Olig001 ASPA - Myrtelle receives Regenerative Medicine Advanced Therapy (RMAT) status for Canavan disease in USA

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