ZURICH, Sept 21 (Reuters) - A European Medicines Agency panel recommended approval of Spark Therapeutics’ gene therapy for blindness, a move that also boosts Swiss drugmaker Novartis that bought the rights to one of the world’s costliest treatments outside the United States.
The Committee for Medicinal Products for Human Use (CHMP) recommended here/news_and_events/news/2018/09/news_detail_003025.jsp&mid=WC0b01ac058004d5c1 voretigene neparvovec, approved as Luxturna in the United States, for patients suffering from inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder that causes vision loss and usually leads to blindness.
The European Commission must approve the drug before it goes on sale, but it normally follows the CHMP recommendation.
Novartis is expanding into gene and cell therapy for rare diseases. Beyond its pact with Spark, the company’s Kymriah CAR-T therapy is approved for advanced blood cancer, while a prospective gene therapy for spinal muscular atrophy (SMA) came with its $8.7 billion purchase of AveXis earlier this year.
And Novartis Chief Executive Vas Narasimhan is on the hunt for more.
“We have three, but of course we’re also looking for more opportunities, with a focus on certain therapy areas where we already have a presence,” Narasimhan told Reuters in a recent interview.
“That’s something we’re actively looking at,” he said.
The treatments have more in common than being at medicine’s cutting edge. They are also very expensive, with Kymriah running $475,000 and the prospective SMA drug potentially commanding more than $1 million, analysts have said, for a one-time treatment.
Spark’s Luxturna is no exception, with $850,000 price tag in the United States giving it the highest list price for any drug, at least for now.
The blindness treatment, approved in December by the Food and Drug Administration, is the first U.S.-approved gene therapy for an inherited disease.
It had only $6.7 million in sales in the first half, but some analysts see it rising to more than $300 million by 2022, according to Thomson Reuters forecasts.
When Novartis licensed the Spark drug earlier this year, it agreed to pay $105 million upfront, up to another $65 million in milestone payments based on European regulatory approval and future sales, and royalties on sales.
Despite early sales of Luxturna, Spark has faced challenges in other areas of its gene therapy development program.
In August, the company said two out of 12 patients showed an unfavorable immune response when treated with a higher dose of Spark’s hemophilia drug SPK-8011, causing its shares to tumble.
The stock is up about 13 percent for the year but is well off highs from early July. Novartis stock is down 1 percent in 2018. (Reporting by John Miller in Zurich and Muvija M in Bengaluru; Editing by Sriraj Kalluvila)