Full data from the Gem-3 trial of Krystal Biotech’s Vyjuvek seem to confirm that the dystrophic epidermolysis bullosa project has a good chance of approval. The study was already known to be a success on its primary and secondary wound healing endpoints, and this weekend a closer look at durability was provided at the American Academy of Dermatology meeting. On a call today Hubert Chen, Krystal's head of clinical development, said the six-month trial allowed the company to demonstrate a consistent benefit over time with retreatment; unlike many gene therapies it is possible to redose Vyjuvek, although the company expects a patient’s usage to decrease over time. The FDA’s requirements on durability have been met with these data, Mr Chen said; a BLA is planned for the second quarter, and a filing in Europe will happen later this year. EB is a highly complex condition, however, and regulatory outcomes are particularly hard to call when small companies are involved – the CRL for Krystal's rival Amryt is a case in point. Krystal is already turning its attentions to launch, however, and with $500m in the bank it has the funds to press on should approvals be granted.
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