Santhera receives negative CHMP opinion and will appeal decision

Swiss specialty pharmaceutical company, Santhera Pharmaceuticals, has received a negative opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) for its revised marketing authorisation application for Raxone in Duchenne muscular dystrophy (DMD).

The company, which gave its oral explanation to the CHMP earlier this week to support the clinical relevance of the existing data in the proposed indication, is planning to appeal this negative opinion decision. Uncertainties over whether the DELOS trial supplied sufficient evidence of efficacy were raised by the CHMP, leading it to adopt a negative opinion of the Type II variation of the company’s existing marketing authorisation for Raxone.

“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, PhD, 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.”

Currently, the therapy is indicated to slow the decline of respiratory function in patients with DMD who are not taking glucocorticoids. The Type II variation would include patients who were previously treated with glucocorticoids or in whom glucocorticoid treatment is not tolerated or is considered inadvisable.