RegulatoryCompany release

MeiraGTx to file bota-vec MAA with EMA in December, FDA BLA to follow in Q1 2027

🇺🇸 MeiraGTx Holdings plc (MGTX) · Thu 12 Nov 2026

MeiraGTx says its dossier for the X-linked retinitis pigmentosa gene therapy is complete, with a European filing planned for December 2026 and a US submission early next year.

MeiraGTx Holdings plc has said the marketing authorisation application (MAA) for bota-vec (botaretigene sparoparvovec), its gene therapy for X-linked retinitis pigmentosa (XLRP), is complete. The company plans to submit it to the European Medicines Agency in December 2026. The update came with its third-quarter 2026 results on 12 November 2026.

The full Biologics License Application (BLA) to the US Food and Drug Administration is expected to follow in the first quarter of 2027, according to the company. The sequencing means the EU filing is due first, with the US submission coming a few months later.

MeiraGTx also gave an update on other programmes. The AQUAx2 pivotal Phase 2 trial of AAV-hAQP1 (AAV2-hAQP1) in radiation-induced xerostomia remains on track to read out in mid-2027. A Phase 3 study of AAV-GAD in Parkinson's disease is planned to begin in the first quarter of 2027.

The company further noted a licence agreement with Lilly for AAV-AIPL1, which carries a $75 million upfront payment. No further terms of the deal were given in the information available.

What it means for your team

  • Regulators should expect a bota-vec MAA to reach the EMA in December 2026, followed by a US BLA in Q1 2027; validation and review timelines will start only after submission.
  • Teams tracking advanced therapy medicinal products (ATMPs) in inherited retinal disease should add XLRP to their watchlists for 2027 regulatory activity.
  • Mid-2027 AQUAx2 data and the planned Q1 2027 Parkinson's Phase 3 start are further milestones for gene therapy pipeline monitoring.

Company's press release (SEC filing): MeiraGTx Holdings plc ↗

Article written by VigiEye from the official source (MeiraGTx Holdings plc). Claims are the company's own. AI-assisted; check the source before acting.

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