The application for use of LUMEVOQ gene therapy to treat vision loss in patients with Leber Hereditary Optic Neuropathy (LHON) due to a mutated ND4 mitochondrial gene was submitted in September, and the procedure was formally initiated on
'We are delighted that the formal review is now underway and look forward to clarifying any questions that the EMA's scientific committees may have,' commented
LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new ND4 LHON patients in
Lenadogene nolparvovec (tradename: LUMEVOQ) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which was specifically developed for the treatment of LHON associated with mutation in the ND4 gene. GenSight submitted the MAA based on the benefit-risk balance established by results from a Phase I/IIa study (CLIN-01), two pivotal Phase III efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow up study of RESCUE and REVERSE (CLIN 06 - readout at Year 3 post injection), supported by a statistics-based indirect comparison methodology to establish the gene therapy's efficacy compared to natural history.
The next major administrative milestone step in the review is a pause at Day 120, at which point the Committee for Advanced Therapies will issue a first round of questions and the Company responds as well as submitting information that had been pre-agreed with the agency.
The Company is also working towards submitting LUMEVOQ's Biologics License Application (BLA) to the
About
About LUMEVOQ (GS010; lenadogene nolparvovec)
LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in
About Leber Hereditary Optic Neuropathy (LHON)
Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in
Contact:
Thomas Gidoin
Tel: +33 (0)1 76 21 72 20
Email: tgidoin@gensight-biologics.com
(C) 2020 Electronic News Publishing, source