MeiraGTx Holdings plc announced on November 12, 2026, that it has completed the marketing authorisation application (MAA) dossier for botaretigene sparoparvovec (bota-vec) and intends to submit it to the European Medicines Agency (EMA) in December 2026. The company also stated it expects to submit a full Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the treatment of X-linked retinitis pigmentosa (XLRP) in the first quarter of 2027. MeiraGTx acquired full rights to bota-vec from Johnson & Johnson in May 2026.
The filing details that XLRP is a rare inherited retinal disease affecting over 20,000 patients in the U.S. and EU, characterized by early onset and progressive blindness. The Phase 3 LUMEOS study involved 95 patients treated bilaterally. The company reported that bota-vec has received Fast Track and orphan drug designation from the FDA, as well as Priority Medicines (PRIME) and orphan drug designation from the EMA.
Regarding financial results for the quarter ended September 30, 2026, MeiraGTx reported cash, cash equivalents, and restricted cash of $186.8 million, a significant increase from $17.1 million in the prior year period. Service revenue for the quarter was $1.6 million, driven by contract manufacturing services provided to Eli Lilly and Company under the AAV-AIPL1 collaboration agreement. The company noted that with the $95.0 million upfront payment due from its partner Hologen, it believes it has sufficient capital to fund operations through the second half of 2028.
Operational updates include the appointment of John Knighton as Executive Vice President of Global Manufacturing and Supply Chain and Alan M. Sebulsky to the Board of Directors. Additionally, the company highlighted the upcoming 12-month data readout from the pivotal Phase 2 AQUAx2 study for radiation-induced xerostomia in mid-2027, which has received Breakthrough Therapy Designation from the FDA.
