Spark's Gene Therapy Hemophilia Data Give Investors The Jitters
Company explains how it will design upcoming Phase III hemophilia A study of SPK-8011 for success, but some analysts still think it's risky business.
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During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.
As updated Phase I/II results for BioMarin's hemophilia A and Spark's hemophilia B gene therapies were presented on May 22, it was revealed that FDA plans to make hemophilia the first gene therapy indication eligible for accelerated approval based on a surrogate endpoint.
Novel reimbursement scheme allowing payers to spread out the cost of an expensive gene therapy over time may get test drive with Spark's Luxturna before being used for big-market treatments for hemophilia.