From Nature Reviews Drug Discovery Feb 2013
Breakthrough drug programme debuts
Vertex receives the first 'breakthrough drug' designations, and application volume is on the rise.
The lowdown: With the introduction of the US Food and Drug Administration Safety and Innovation Act (FDASIA) last summer, the FDA was committed to creating a 'breakthrough drug' programme to expedite the development and review of drugs for serious or life-threatening diseases that may offer substantial improvement over existing therapies, based on preliminary clinical evidence. “The programme is to really highlight drugs that are game changers,” said John Jenkins, Director of the Office of New Drugs at the FDA's Center for Drug Evaluation and Research. Sponsors who are granted the designation are entitled to increased interaction with the FDA to ensure the drug development programme is streamlined and clinical trials are optimized for efficiency.
The first beneficiaries of the programme are Vertex's ivacaftor and its combination of VX-809 plus ivacaftor, the company disclosed at the J. P. Morgan meeting in San Francisco. Ivacaftor is already approved for cystic fibrosis in patients with at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, but the new designation could accelerate its approval into other patient groups as well.
As of mid-January, the agency had received a total of 16 applications for breakthrough drugs. Only the 2 from Vertex had been approved as Nature Reviews Drug Discovery went to press; 3 others had been rejected and 11 were still pending.
Draft guidance on the programme is due to be published by the end of 2013. But already, one key element is becoming clear: “[Companies] are really going to have to adjust their manufacturing plans,” says Jenkins. A company might hypothetically be able to complete the clinical trials needed to support an application in 1 year, he explained, but this is of little benefit if it does not plan to build a production plant for 3 years.
