The often-troubled field is showing promise, and the extra resources that breakthrough designation will bring to two candidates could be what gene therapy needs to move from the theoretical to the marketable.
The field of gene therapy is heating up again, with candidates advancing toward late-stage trials and eventual submission to FDA. The extra resources and attention provided by FDA's breakthrough therapy program could help nurture the whole field through the inevitable regulatory challenges that the novel and highly complex products will bring.
Spark Therapeutics Inc. is at the forefront of gene therapy breakthrough designations (BTDs), with two of the five BTDs...
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Brazilian regulator Anvisa is relying on the decisions of its foreign counterparts as a means of ensuring more quality medicines are made available in the country.
The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.
The UK regulator approved 16 medicines containing a new active substance in the first half of this year, while the European Commission issued marketing authorizations for 20 NAS-containing drugs.
Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.
