Santhera ‘surprised’ by European rejection of DMD drug
pharmafile | September 18, 2017 | News story | Sales and Marketing | Duchenne Muscular Dystrophy, biotech, drugs, pharma, pharmaceutical, santhera
Santhera Pharmaceutical has announced the EMA’s CHMP rejected its treatment for Duchenne muscular dystrophy (DMD) but revealed that it planned to appeal the decision. The rejection came as a surprise to investors, as well as the company, with shares plummeting by over half their value on the announcement.
The treatment that Santhera was hoping to expand the indication of was Raxone (idebenone), a therapy that is currently approved for Leber’s hereditary optic neuropathy (LHON) in Europe. However, in a decision that echoed the FDA’s previous ruling on its application for Accelerated Approval, the CHMP suggested that the Phase 3 data that had been submitted as the basis for approval was not sufficient.
“We are surprised and disappointed by the opinion of the CHMP. Data from the phase III DELOS trial demonstrated statistically significant and clinically relevant evidence that Raxone slows the decline of respiratory function, and reduces the risk of bronchopulmonary complications and hospitalization in patients with DMD not using glucocorticoids,” said Thomas Meier, CEO of Santhera. “These patients in the respiratory decline stage currently have no treatment options, and because we are confident that they could benefit from treatment with Raxone, we plan to appeal this opinion and seek re-examination.”
For Santhera, the rejection for the treatment could feel familiar – after it also struggled to gain approval in LHON, with an initial rejection before it was finally approved. The main issue the company faces now is a lack of investor confidence, with more than $200 million wiped from its market value after the rejection.
The precipitous drop in value is an indication of the confidence that investors had held that the treatment would be accepted. Raxone was one of the few treatments to be given to UK patients as part of its Early Access to Medicines Scheme, based on the Phase 3 data, and it was widely assumed that this was an expression of confidence in the medicine.
There is further trial data to come on the medicine, with a Phase 3 trial (Sideros) currently on-going. However, results from this trial are not expected until 2019 –leaving a huge delay before being able to again approach the FDA and, potentially, the EMA.
Ben Hargreaves
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