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Santhera Pharma slammed with EU denial of orphan drug

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Santhera Pharmaceuticals suffered a setback today with the rejection of its key experimental drug for a genetic cause of blindness. European Union reviewers denied the Swiss specialty pharma their support for approval of Raxone over concerns about the small number of patients studied during clinical development.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) dealt Santhera the bad news, the company said, noting that a narrow majority rejected the drug for treating Leber's Hereditary Optic Neuropathy. Santhera, as one would expect, is pushing for EU reviewers to reconsider its treatment based on the clinical need the drug addresses.

Yet that didn't stop investors from scrambling. Santhera's shares fell hard on the disappointing news, and Bloomberg reported that the share price sank as much as 31% to 3.30 Swiss Francs.

"Since there were no concerns expressed about the safety of Raxone and the observed treatment benefit on visual acuity in these patients was clinically relevant and consistent with the published literature, we believe that we should be allowed to address the remaining concerns through further confirmatory clinical work to be conducted post-approval," Santhera CEO Thomas Meier said in a statement.

Santhera is among a bevy of biotechs around the world banking on development of drugs for rare or orphan diseases. Raxone would be the first drug for LHOP, which is causes by mutations in mitochondrial DNA and swiftly robs patients, mostly men, of their sight months after symptoms crop up.

- here's the release
- see Bloomberg's article

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