Essai REGAIN Ph III : Amélioration des données secondaires chez des patients MG traités par SOLIRIS

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Essai REGAIN Ph III : Amélioration des données secondaires chez des patients MG traités par SOLIRIS

Message par Pboulanger » 10 juil. 2016 10:14


Lu sur ... th-soliris

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Secondary data show improvement in gMG patients treated with Soliris

US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) has presented new secondary efficacy endpoint data on Soliris (eculizumab) in the treatment for refractory generalized myasthenia gravis (gMG) showing patient improvement at week 26.

Last month, Alexion reported that its Phase III REGAIN study of the drug failed to meet its primary endpoint, but the latest results showed that 18 of 22 pre-defined endpoints and pre-specified analyses in the study achieved p-values <0.05. The new data were presented at the Hot Topics session of the 14th International Congress on Neuromuscular Diseases (ICNMD) in Toronto.

New secondary efficacy endpoint data presented included change from baseline in Myasthenia Gravis Composite (MGC) score at week 26, which achieved a p-value of 0.1026, and change from baseline in the 15-item Myasthenia Gravis Quality Of Life (MG-QOL15) at week 26, which achieved a p-value of 0.0281, both measured by a worst-rank analysis. A pre-specified sensitivity analysis of the MGC and MG-QOL15 endpoints using repeated measures from baseline to week 26 achieved p-values of 0.0134 and 0.0010, respectively.

Aiming for new indication to be approved

Alexion is hoping to gain an additional indication of myasthenia gravis, a rare neuromuscular disorder, for it ultra-orphan drug Soliris, which has come up for criticism for being hugely expensive; it costs up to $560,000 per patient a year. In the first quarter of 2016 net product sales of Soliris were $665 million compared to $600 million in the like 2015 period, accounting for the vast majority of Alexion’s total revenues of $701 million for the quarter. The company is forecasting full-year 2016 sales of Soliris in the range of $2,835 to $2,875 million

Soliris is currently approved in the USA (2007), European Union (2007), Japan (2010) and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. It is also approved in the USA (2011), EU (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels).

“Although the REGAIN study narrowly missed its primary endpoint, the additional data presented today suggest a magnitude of effect of eculizumab in refractory MG patients across four separate scales of disease severity that is unprecedented in my clinical investigation experience,” said James Howard Jr, Distinguished Professor of Neuromuscular Disease, Professor of Neurology, Medicine & Allied Health, and Chief, Neuromuscular Disorders Section, The University of North Carolina School of Medicine. “These findings are particularly meaningful given the urgent need for a first-ever therapy with the potential to have a transformative impact on patients with refractory MG, who continue to face disabling limitations in their daily lives, including difficulty walking, talking, swallowing, and breathing normally,” he noted.

“The MG Community has been waiting for the full results from the REGAIN trial with great interest,” said Nancy Law, chief executive of the Myasthenia Gravis Foundation of America (MGFA), adding: “There are currently no FDA-approved disease modifying therapies for MG, which means that doctors and patients must often resort to trial and error with medications approved for other conditions to try to manage symptoms. For all too many with MG, the currently available treatment approaches are inadequate in efficacy, or have intolerable side effects. People with MG need more and better treatment options, especially for those with refractory generalized MG, whose extreme muscle weakness can be devastating and even life-threatening. We look forward to advocating for and supporting patients with refractory MG who have a tremendous unmet need.”


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