ViewPoints: FDA throws weight behind Kalydeco

The FDA approval of Vertex’s cystic fibrosis (CF) therapy Kalydeco (ivacaftor) – over two months ahead of schedule – caught many by surprise. The decision both demonstrates decisiveness on the part of the regulator and a successful multi-faceted approach by Vertex to support approval.

Insight & Analysis

Accelerated reviews Expedited approvals by the FDA came back into focus last week with clearance for Roche’s skin cancer treatment Erivedge (vismodegib) and Vertex’s Kalydeco. The agency has faced criticism in the past for both acting too slowly and too rapidly, withdrawal of Roche’s Avastin (bevacizumab) in November acted as a recent catalyst for such debate.

Minimal scrutiny in this case Kalydeco, however, will not face similar scrutiny according to Decision Resources analyst Richa Mukherjee, given that it is the first therapy to treat the underlying cause of CF and provides much simplified oral dosing.[2] Those looking for controversy are therefore likely to turn their attentions to the price, which at an annualised rate of $294,000 per patient positions Kalydeco as one of the world’s most expensive medicines. The price is justifiable, according to Vertex, as it reflects the value brought to a small subset of CF patients eligible for treatment.

Specific population The highly targeted nature of Kalydeco – designed for 4 percent of the CF population – is, according to Mukherjee – a key facet in both demonstrating the efficacy of the drug and supporting rapid FDA approval. Speaking to FirstWord, she cited both the "clean and clear" data facilitated by use among patients with a specific mutation and the various engagements between Vertex, physicians, patient advocacy groups and the FDA to share data and support approval, concluding that this acts as a strong model for submissions of this type.

Opinion

Decision Resources analyst Richa Mukherjee: "If developers can target a particular population it’s very clean in terms of the clinical trial. Even though the recruiting might be harder, in terms of getting results they are clear to the regulatory agency."

Mukherjee: "The FDA will have relied on a lot of information by the company via, for example, patient advocacy groups. In this case the Cystic Fibrosis Foundation has done an amazing job in supporting drug development. There will be a constant flow of information with the FDA which I think makes it easier to get approval like this. Maybe this could be a model for how you could get something quickly on the market."

Sanford Bernstein analyst Geoffrey Porges: "The FDA is trying to demonstrate a willingness to move quickly on medicines that make a big difference and this is a big difference maker."

 

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