EMA, Gene Therapy

Novartis drums up EU launch plans as CHMP gives thumbs up to Spark’s gene therapy Luxturna

Nine months after the FDA gave its landmark OK for Spark Therapeutics’ trailblazing gene therapy Luxturna, European regulators have signaled that they are ready to greenlight the vision loss treatment, too.

Paul Hudson

The team at Spark $ONCE isn’t the only ones celebrating the positive opinion from the Committee for Medicinal Products for Human Use: Novartis, which grabbed ex-US rights to Luxturna earlier this year in a $170 million deal, will be commercializing the therapy once it’s approved in Europe.

“Today’s positive CHMP opinion represents a significant step in our journey toward advancing potentially life-changing cell and gene therapies in ophthalmology,” said Paul Hudson, CEO of Novartis Pharmaceuticals, in a statement. “We look forward to working with Spark Therapeutics and the EMA to establish access and reimagine care for people in the EU who face the threat of total blindness from this inherited retinal disease.”

Jeffrey Marrazzo

While (unlike in the US) Luxturna won’t be the first gene therapy approved in the EU — GlaxoSmithKline and uniQure beat them to it with their therapies for “bubble boy syndrome” and familial lipoprotein lipase deficiency — Novartis is hoping it would be the first one with a real commercial prospect.

Price would be key here, especially as the pharma giant faces with a group of single payer operations with considerable leverage over pricing.

Some analysts have also expressed concerns about the small market: Luxturna is designed as a one-time treatment for a rare form of vision loss (which can eventually result in retinal blindness) caused by mutations in the RPE65 mutations, which affect 1 in 200,000 people.

In the US, Spark CEO Jeff Marrazzo has painstakingly laid out a pricing model in hopes of convincing payers to cover the $850,000 procedure — $425,000 per eye — for a tiny group of under 2,000 potential patients, with fewer than 20 new cases per year. The biotech booked $6.7 million in net sales for the first six months of the year, but it’s still too early to tell how that’s working.

The positive CHMP opinion is based on a data package that included a total of 43 patients with inherited retinal disease caused by RPE65 mutations. A marketing authorization from the European Commission is expected to follow within two months.


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