What is LUXTURNA or Voretigene Neparvovec-RZYL?
LUXTURNA is the trade name for voretigene neparvovec-rzyl. It is developed by Spark Therapeutics, now licensed to Novartis. LUXTURNA is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy – As per the US FDA. LUXTURNA is ideal for patients with viable retinal cells; the same has to be clinically determined. Luxturna happens to be the first directly administered gene therapy approved by the FDA. It is unique because of its mechanism of targeting a particular disease caused by a specific gene mutation. The entire therapy is developed through recombinant DNA techniques and deliver normal human RPE65 gene to the retinal cells. Restoration of vision is the targeted outcome of this therapy.
LUXTURNA – FDA Approved Cellular and Gene Therapy Product
LUXTURNA received its US FDA approval in December 2017. A phase III study with 31 participants was the basis for its approval. Its safety and efficacy is determined through a clinical development program that involved 41 patients. They aged between four and forty four years during the assessment period. A patient or participant’s ability to navigate an obstacle course at various light levels is the parameter for evaluation. Patients experienced significant improvement with LUXTURNA. Long term safety analysis and post marketing studies are expected to provide more insights. LUXTURNA was granted Orphan Drug and Breakthrough Therapy designations by the FDA. More information can be found in the US FDA Press Release.
How does treatment with LUXTURNA (voretigene neparvovec-rzyl) work?
Cellular and Gene Therapies are different, some are very complex and sophisticated. Many such therapies are designed with restorative purposes and are not tested in a large number of patients. The entire therapy may be customized for a specific patient in some cases. LUXTURNA is not a completely personalized therapy; it is supplied in a 0.5-mL extractable volume in a 2-mL single-dose vial. It is administered as subretinal injection. The recommended dose of LUXTURNA for each eye is 1.5 x 1011 vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL. It is a onetime therapy; patients above 12 years are eligible for the same. A specific genetic test is done to determine if a patient is the right candidate for this gene therapy. Refer to the LUXTURNA Prescribing Information for more specific details.
Patients and caregivers can visit luxturna.com for more information and insights. It has comprehensive information and provides a feature to find a specialist in the US. The website is exclusive for the US patients.
LUXTURNA in India – Is it possible?
Right now there is no enough clarity regarding access to LUXTURNA India. Novartis has its registered office in India; queries can be sent to their official representatives. You can email us nhs@noelgenomics.com and register your interest regarding this therapy. We are committed to share authentic access information. We intend to assist Indian patients looking for advanced therapeutic solutions.
