J.P. Morgan Notebook Day 2: Bristol Humbled By Competition, Sanofi’s Sarilumab Ready For Review, Justifying Spinraza’s Price And More
Daily round-up of news and notes from the 2017 J.P. Morgan Healthcare conference in San Francisco. Bristol-Myers CEO humbled by PD-1 competition in lung cancer; Sanofi’s sarilumab manufacturing issues resolved; Biogen, Ionis discuss SMA drug’s high cost; Shire happy with Xiidra launch to date; Sarepta says more than 250 patients have started Exondys treatment; J&J’s Gorsky on innovation and pricing; and Amicus reveals US approval strategy for migalastat.
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Exondys 51 has been met with recommendations for restrictions by many insurers, but after consultations with experts and Sarepta execs coverage is expanding.
The WORLD Symposium Feb. 13 to 16 in San Diego offered everything from basic research through late-stage clinical data in lysosomal diseases, including ArmaGen's blood-brain barrier-crossing treatment for Hurler syndrome and updates from ongoing development programs from rare disease specialists, such as Genzyme, Amicus and Protalix.
Shire’s Perry Sternberg and Phil Vickers are optimistic about recent launches and products in late stages of development in key rare disease and specialty therapeutic areas, many of which come from Baxalta and other high-profile acquisitions.