On Thursday, an expert panel gathered by the USFDA held an advisory committee meeting ahead of the agency’s approval decision on Spark Therapeutics’ new treatment “Luxterna”. FDA regularly hold this type of open hearings with advisory committees when completely new drugs are in the process to become marketed products. So what was so special about this particular committee meeting?

Luxterna is the first gene therapy treatment to come under FDA review whereby a genetic disorder gets corrected by the insertion of a healthy gene. Children born with this disorder develops gradually worsening eyesight symptoms, particularly loss of night vision, and often complete blindness before adulthood. In this case, Luxterna corrects mutations in a gene that causes damages to the retina in the eye. A real breakthrough by the use of gene therapy.  

Spark utilizes engineered viruses as an agent to insert the correct version RPE65 directly into the eye. Clinical studies of Luxterna showed maintained vision in over 90 percent of patients and many experienced improved vision from the once-only treatment. In addition, the treatment did not raise any safety concerns. It was therefore, not a surprise that the 16 experts in the panel, after a vote, gave their unanimous recommendation to the FDA to approve Luxterna. Spark Therapeutics was up some 5% in the aftermarket following the panel vote.

With the final FDA decision expected in January,  Spark is now working on duplicating their approach in other genetic eye diseases. However, the company is also attempting to correct the genes causing hemophilias, an area where a few other biotech company also have gene therapy programs ongoing with promising results so far. One of these, a small cap company Voyager Therapeutics also part of the AALS portfolio, presented not long ago very promising gene therapy data in patients with Parkinson’s disease. Safe to say, the team believes we will hear a lot more from the field of gene therapy going forward. 

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Arctic Aurora LifeScience is an equity fund investing in global biotechnology and pharmaceutical companies. The fund is run by former portfolio manager in the Swedish AP Fonder, Ulrica Bjerke, and Dr. Torbjørn Bjerke, both with 20 years of experience from the industry. Daniel Bolanowski fills the role as anlyst on the team with a MSc in both Biotech and Economics. Arctic Aurora LifeScience was launched in May 2016 with both hedged and un-hedged share classes.

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