Renewed Momentum for XLRP: MeiraGTx Targets Rapid Approval Path for bota-vec following reacquisition from Johnson and Johnson
It has recently been announced that Meira-GTx, who originally developed bota-vec and collaborated with J&J during its early clinical trials, have entered into an asset purchase agreement with J&J to acquire bota-vec and progress the program towards global regulatory filings for approval.
In a previous article from July 2025, we shared the news that Johnson and Johnson’s (J&J) development of botaretigene sparoparvovec (bota-vec), a gene therapy for X-linked Retinitis Pigmentosa (XLRP), had come to an abrupt halt. This was due to the reported failure to reach the target primary endpoint in a phase 3 clinical trial. At the time, the outcomes of key decisions by J&J were unknown and the direction for bota-vec’s further development remained unclear, despite previous positive findings causing excitement within the inherited retinal disease community. You can read our previous article on this here.
It has recently been announced that Meira-GTx, who originally developed bota-vec and collaborated with J&J during its early clinical trials, have entered into an asset purchase agreement with J&J to acquire bota-vec and progress the program towards global regulatory filings for approval.
“We are extremely pleased to have reacquired bota-vec for the treatment of XLRP. This is a unique opportunity to gain an asset at this stage in development with data supporting a meaningful benefit in patients with no alternative treatment, many of whom are waiting for this life changing therapy and hoping for expeditious approval.” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx
What does this news mean?
Despite failing to meet the primary endpoint in the phase 3 LUMEOS trial, data from secondary endpoints showed that participants experienced various clinically meaningful benefits and the safety profile remained consistent with previous trials, as discussed in our previous article. Along with this data, MeiraGTx had also heard compelling evidence from investigators involved in the trial about the participant benefits.
Meira-GTx is in a favourable position to move quickly towards filing for approval and bota-vec has been granted several Fast Track and Orphan Drug designations from various regulatory bodies which are designed to expedite the normally lengthy review process. Meira-GTX intends to file for approval as quickly as possible in the U.S, EU, UK and Japan with the potential for a commercial launch in 2027 in one or more of these regions. Although regulatory and subsequent NHS approval is by no means guaranteed, this is an exciting step forward for our XLRP community, and we look forward to hearing the updates from MeiraGTx over the next year.
Kate Arkell, Research Development Manager at Retina UK, said “We are excited to hear that MeiraGTx is taking up the baton to progress bota-vec through regulatory review. Retina UK will support MeiraGTx wherever possible to facilitate future access to this promising therapy for our X-linked RP community.”