The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.
But mixed data and a previous failure provide reasons to be cautious.
But a lacklustre pipeline could stoke the need for deals.
The group will need longer-term data to justify its huge valuation.
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
One-year data look encouraging, but etranadez will now not be filed until 2022.
The whole haemophilia gene therapy field will be hoping that a case of liver cancer in etranacogene dezaparvovec’s pivotal trial is not treatment related.