The Food and Drug Administration recently announced the first ever approval of a directly administered gene therapy in the United States. Spark Therapeutics one-time gene therapy Luxturna is indicated for the treatment of vision loss caused by biallelic RPE65 mutation-associated retinal dystrophy, and is the first and only pharmacologic treatment for an inherited retinal disease. Given the strength of the pipeline, the approval of Luxturna has intensified competition amongst companies developing gene therapies, and the commercial success of the treatment could have serious implications for therapies still in development.
Historically the pricing of approved gene therapies has presented major issues, ultimately leading to a lack of demand for both UniQure’s Glybera and GSK’s Strimvelis, both of which were approved in Europe and will now serve as a cautionary tale for Spark as they seek to commercialise Luxturna, which has been given a price tag of $425k per eye. In addition to the well-documented pricing issues associated with gene therapies, a recent manufacturing crisis has grabbed the attention of the biopharmaceutical community, prompting analysts to question whether this is the beginning of a successful journey or an emerging manufacturing disaster.