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SMA children say " We need olesoxime. Now ! "
2016 - TIME for DISAPPOINTMENT January 2016, ROCHE restarts a phase II clinical trial (NCT02628743) to evaluate long term safety, tolerability, and effectiveness of olesoxime in SMA Patients. A very few number of SMA patients will be included in this open-label trial as it will only concern people who were previously enrolled in the Trophos SMA trials (2012/2013). Estimated study completion will end in august 2020. This announcement came as a blow for all SMA families.
- Why does it take so long ? clinical trials for olesoxime is a long story : phase Ib took place in 2008-2009 ; phase II/III already took place in 2011-2013 and now ROCHE announces that olesoxime goes back to a new phase II (two) trial.
- When will olesoxime be effectively available to people with SMA ?
2014 - TIME for HOPE Results from a Trophos pivotal phase II/III clinical trial with olesoxime in SMA (2012-2013) showed a beneficial effect on the maintenance of neuromuscular function in individuals with Type II and non-ambulatory Type III SMA, as well as a reduction in medical complications associated with the disease. These data were first presented in April 2014 at the annual meeting of the American Academy of Neurology (AAN), Philadelphia, PA, USA. Analysis of the data from the pivotal trial of olesoxime provided further evidence of its neuroprotective effect, with a statistically significant impact on maintenance of motor function. Moreover, US and EU regulatory authorities have granted orphan drug designation to olesoxime.
Olesoxime had the potential to be the first ever treatment approved especially for SMA patients.
SMA and OLESOXIME Early 2015 ROCHE Pharmaceutics acquired Trophos, a privately held biotechnology company based in France. During 15 years (1999 - 2014), Trophos’s proprietary screening platform generated olesoxime (TRO19622), which is being developed for spinal muscular atrophy (SMA), a rare and debilitating genetic neuromuscular disease that is most commonly diagnosed in children. It is the number one genetic cause of death in children under the age of two. There is currently no effective and no approved treatment for SMA.
EXPRESS REQUESTS "Families of SMA France" understand that there are many steps before EMA (European Medicines Agency) and FDA (Food and Drug Administration - USA) approvals. According to ROCHE current timeline, the company will submit its regulatory files (EMA/FDA) during the first half of 2017. Then, a final marketing authorisation could take place only in 2018, 2019 or 2020. This very late delay is unacceptable.
Olesoxime trial progress :
phase Ib : 2007/2009
phase II/III : 2010/2013
phase II : 2016/2020
In our view, the key step was reached at the end 2013, when the data showed that SMA patients treated with olesoxime were able to maintain motor function over the two-year period of the first study (2012/2013) and that typical healthcomplications associated with SMA occurred less frequently than in patients treated with a placebo, leading to better well being. Now SMA people need olesoxime as soon as possible.
Each day that passes without a treatment leads to deterioration of muscle functions for SMA patients.
February 15th, 2016
Waiting for olesoxime time and time again...
"Families of SMA France" asks ROCHE to take the fastest route possible to get olesoxime to SMA patients. The olesoxime approval process must be accelerated.
please RT #olesoxime4all_now @ROCHE
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