Approval status
Session cochair Gianfranco De Stefano, MD, department of human neuroscience, Sapienza University of Rome, asked Dr. La Morgia about the current approval status of lenadogene nolparvovec.
She said that it was presented to the European Medicines Agency for approval, but the application was withdrawn earlier this year. The “main criticism” was that bilateral improvement was seen even in patients who received only a unilateral injection.
This is “not easily explainable,” said Dr. La Morgia, although it was found that the viral vector was present in the uninjected eye.
There was also a question regarding the heterogeneity of the patient data, which it is hoped will be addressed in future clinical trials.
Commenting after the session, De Stefano said in an interview that the results are “very interesting” and “very promising.”
He pointed out that idebenone may be the only currently available therapy for LHON, but it is “not very effective, and it’s something you give the patient just for the sake of doing something” in light of the possibility that he or she might have “even a small improvement” in eyesight.
However, he believes that lenadogene nolparvovec is a long way from becoming available in the clinic, primarily because longer follow-up is required to determine whether just one injection is enough.
“It may be likely that this gene therapy does not have a long-lasting effect,” he explained.
Currently, the longest follow-up is just 2 years; “I don’t know if there will be a need for repeat injection,” De Stefano said.
No funding was declared. Dr. La Morgia has relationships with Chiesi Farmaceutici, GenSight Biologics, Regulatory Pharma Net, Thenewway, Santhera Pharmaceuticals, First Class srl, Biologix, Stoke Therapeutics, and Reneo.
A version of this article first appeared on Medscape.com.