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Latest From Emily Hayes
With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.
Fast US approval of ultra-rare pediatric disease drug Brineura (cerliponase alfa), with an annual list price of $702,000, is a victory for the company, but approval for restricted pediatric population compared to Europe raised questions.
Opdivo first-quarter sales were still strong at $1.1bn, largely driven by lung cancer use and helping to drive overall performance, but potential approval of Merck's competing Keytruda with chemotherapy in first-line lung cancer is right around the corner.
Emerging Company Profile: New technology platform aims to apply directed evolution to identify small molecule candidates, much like large molecules like monoclonal antibodies are developed, which the Calif. start-up thinks can bring oral drugs to new fields.
The third IL-17 inhibitor on the US market will have a $3,500/month list price. But in a crowded, safety-conscious space like psoriasis, it's unclear how low the discounted price will need to go and whether competitive pricing would be enough to sway prescribers.
Amgen's FOURIER outcomes study of Repatha supports LDL-lowering in high risk patients, but price may have to come down dramatically to spur wide use of the PCSK9 inhibitors with the subjective condition of statin intolerance.