EMA confirms negative stand for Santhera’s Raxone extension application in Duchene

Santhera Pharmaceuticals will still remain without extension application for Raxone in Duchenne muscular dystrophy (DMD) in Europe, as expected. The company said on Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on the Type II extension application for it following a re-examination procedure.

To remind, Santhera in September 2017 received the negative opinion on this matter, and it then said it would appeal about the decision.
Earlier this week, it said it expects the negative opinion.

According to Santhera, the CHMP said it can’t approve Raxone in DMD, applied as a Type II variation of the existing marketing authorization, but it said that Santhera can offer additional data to link the positive treatment effects on respiratory functions.
The positive outcome of the Phase III DELOS trial was acknowledged, Santhera noted.

“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. The CHMP acknowledged the unmet medical need in this patient population,” said Thomas Meier, PhD, CEO of Santhera.

He added that the company will remain fully committed to realizing the potential of Raxone in treating patients with DMD.

Jon Hastie,  PhD and CEO of DMD Pathfinders, a user led organization of adults with Duchenne,  said that there is an urgent need to find a treatment to slow the decline in respiratory function in individuals who do not take steroids as there is no other treatment option available. “I’m glad that Santhera will continue in their efforts to make Raxone available to patients,” he said.