Orphalan Flies Solo In US After Wilson’s Disease Drug Win
FDA Approves French Firm's Cuvrior
Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."
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Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.
PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.
While Orphazyme's attempts to get approval for arimoclomol failed miserably, new owner KemPharm is confident it can get the oral heat shock protein amplifier across the regulatory finishing line for Neimann-Pick disease type C.