Luxturna Gene Therapy Nears European Market for Inherited Retinal Disease

Luxturna Gene Therapy Nears European Market for Inherited Retinal Disease

For individuals and families living with inherited retinal diseases (IRDs), the prospect of new treatments offers immense hope. The recent positive opinion from European regulators regarding Luxturna (voretigene neparvovec), a gene therapy for a specific form of IRD, marks a significant step forward. This development brings the potential for a new therapeutic option closer to patients in Europe, offering a beacon of progress in the fight against vision loss caused by genetic mutations.

Luxturna, developed by Spark Therapeutics and licensed to Novartis for markets outside the U.S., received a positive opinion from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) in September 2018. This recommendation paved the way for an EU marketing authorization, which is typically a formality within a few months. The European Commission subsequently granted marketing authorization for Luxturna in November 2018. This approval covers all 28 member states of the EU, as well as Iceland, Liechtenstein, and Norway.

Luxturna is a one-time gene therapy designed for adult and pediatric patients experiencing vision loss due to confirmed biallelic RPE65 mutations, provided they have sufficient viable retinal cells. Mutations in both copies of the RPE65 gene can lead to a progressive condition that often results in total blindness. This therapy works by delivering a functional copy of the RPE65 gene to retinal cells via a single injection, aiming to restore the production pathway for a necessary enzyme and improve light detection. Clinical trials, including a Phase 1, its follow-up, and a Phase 3 trial involving 43 participants, provided the data supporting the positive opinion.

This approval is particularly significant as Luxturna is the first gene therapy for a genetic disease to be approved in both the U.S. and the EU. In the U.S., where the drug was approved in late 2017, Spark Therapeutics set the price at $850,000, or $425,000 per eye. While Novartis had not immediately announced pricing details for Europe at the time of the EU approval, the company stated its commitment to working with payers to develop innovative payment models and patient access programs to address the unique aspects of a one-time gene therapy. Novartis indicated that it expected to finalize reimbursement deals in individual European markets in 2019 and 2020. For instance, in Italy, Luxturna's ex-factory price was €360,000, with reimbursement taking effect in January 2021. In Germany, France, and the UK, pricing for Luxturna was reportedly around €295K, €290K, and £269K annually, respectively, with reimbursement decisions expected in 2019 and 2020.

The European approval of Luxturna offers renewed hope for individuals with RPE65-mediated IRD, a condition that previously had no treatment options. The focus now shifts to ensuring equitable access and sustainable reimbursement models across European countries, allowing eligible patients to benefit from this groundbreaking therapy. This development underscores the ongoing progress in gene therapy research for inherited retinal diseases and the commitment of the biotech and pharmaceutical industries to bringing these innovations to patients worldwide.