Kalydeco - Vertex

Cost: $299,592 per U.S. patient per year

Kalydeco, in a word, was revolutionary when it hit the market back in 2012. It was the first treatment approved to address the underlying cause of cystic fibrosis, rather than its symptoms. And it substantially improves lung function in patients with a particular genetic mutation, dubbed G551D, which affects only 4% of the 70,000 CF patients worldwide.

To offset that sales ceiling, Vertex ($VRTX) gave the drug a lofty price tag. But it did set up copay help for eligible patients; when Kalydeco was rolled out, about 60% of patients had commercial insurance, with government programs taking care of the rest. Vertex said its copay assistance program would cover up to $88,000 of privately insured patients' coinsurance or copayments, and that patients would have case managers to help them through payer bureaucracies.

The result? A stellar U.S. launch, followed by a similar one in Europe once the drug hit the continent's major markets. And though the majority of eligible G551D patients in countries where Kalydeco is approved now have access to the drug, Vertex hasn't sapped the med's sales potential. In February, Kalydeco snagged an FDA thumbs-up for 8 additional mutations, which could spell more than $45 million in potential new revenue. And the EMA's European Committee for Medicinal Products for Human Use (CHMP) recommended in June that it do the same.

For more:
Special Reports: Top 15 Drug Launch Superstars - Kalydeco - Vertex Pharmaceuticals | Top 20 orphan drugs by 2018 - Kalydeco
Vertex scores 250 new Kalydeco patients with EMA approval
Vertex eyes another $45 million in sales as FDA swells Kalydeco's patient pool
Vertex asks FDA to expand market for CF drug Kalydeco

-- Carly Helfand (email | Twitter)

Kalydeco - Vertex
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