Regulatory, Results

UK regulators green-light early access to Santhera’s Raxone for Duchenne MD

Thomas Meier, CEO of Santhera

Santhera Pharmaceuticals saw its shares {SIX: $SANN} jump 16% today after the Swiss biotech reported that UK regulators will provide early access to their drug Raxone (idebenone) for use in fighting symptoms of Duchenne muscular dystrophy.

British regulators say that the data indicate that this drug can provide a longterm benefit for boys experiencing respiratory decline and not on or able to take glucocorticoids. They also endorsed the safety profile of the drug.

That all sounds like an encouraging prelude to a CHMP decision on this drug, which is on the market as a treatment for Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease. And Matthew Eckler at RBC says it bodes well for Santhera, indicating an increased likelihood of an approval.

Notes Eckler:

Recall that last month Santhera announced a delay in the expected timing of a CHMP recommendation for Raxone label expansion to Q3 vs. late Q1/early Q2 prior, as a result of a supplementary information request. We continue to view this delay as a wrinkle in the story, rather than fundamentally altering our thesis. We expect a positive CHMP recommendation and label expansion to DMD given: 1) Raxone is the only DMD therapy with a positive Phase III trial; 2) CHMP renewed Translarna’s marketing authorization following failure of its confirmatory study; and 3) Raxone is already marketed in the EU for LHON.

“We’re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” said Thomas Meier, the CEO of Santhera, in a statement.


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