New Medicines

PuldysaDuchenne muscular dystrophy


Licence extension / variation

Development and Regulatory status

Oct 20MHRA removes scientific opinion for Raxone due to discontinuation of EAMS programme at the request of the company [27].
Oct 20EU development has been discontinued [26].
Oct 20EU fling withdrawn due to SIDEROS trial interim analysis [25].
May 20Santhera announces that the EMA has extended the clock-stop in the regulatory procedure. During this period, the company is considering implementing an interim analysis of the SIDEROS study in the hope of demonstrating overwhelming efficacy: if positive, data from this analysis would be included in the review procedure resulting in a CHMP opinion in Q4 2020. It would also accelerate filings for use in patients taking glucocorticoids [24].
Jun 19Trade name in the EU for DMD indication will be Puldysa [23].
Jun 19MHRA renews EAMS opinion for Raxone to treat decline of respiratory function in patients with DMD [22].
May 19Santhera has submitted an application for a conditional marketing authorisation (CMA) for patients with DMD who are not using glucocorticoids [21].
Mar 19Santhera Pharmaceuticals announce intention to file application for Conditional MA in EU for idebenone for treatment of respiratory dysfunction in DMD. Filing will be based on data from PII DELPHI study, long-term DELPHI-Extension study, pivotal PIII DELOS study and SYROS study, a collection of long-term data from patients who completed DELOS study and continued to be treated with idebenone for up to 6-years [20].
Jun 18MHRA renew EAMS opinion for Raxone to treat decline of respiratory function in patients with DMD [19].
Apr 18Santhera informs UK patient organisations that the Commission on Human Medicines (CHM) of the MHRA has reviewed their EAMS approval for idebenone in DMD and has decided to maintain the positive opinion. It considers that the potential benefits outweigh the risks and therefore that continued access via EAMS is justified. The opinion is due for renewal in June 18 and full formal review of the data by the CHM will take place before then. The company hopes that a resubmission will result in a EU product licence in about two years [18].
Feb 18Santhera hopes to re-submit an application to the EMA in 2019. Based on the feedback from CHMP, the company does not expect to have to carry out another trial, but it will have to provide additional data to show the link between respiratory function and overall patient benefit [17].
Jan 18The CHMP has upheld its negative opinion on use of idebenone in DMD: the original grounds for refusing the change in Marketing Authorisation were that although the study data provided showed a difference in Peak Expiratory Flow in favour of the drug, there was no clear improvement in other indicators of breathing function or in muscle strength, motor function or quality of life. After re-examination, concerns over the robustness of the main study remained, particularly regarding its size, protocol changes and early termination, which undermined the robustness of the data. As a result, the CHMP maintained its opinion that the benefits demonstrated did not outweigh the risks in this indication [16].
Oct 17Santhera has requested a re-examination of the CHMP opinion [15].
Sep 17EU negative opinion refusing the addition of a new use of Raxone in patients with Duchenne muscular dystrophy to slow their gradual loss of breathing ability [14].
Jun 17Granted EAMS status in UK [12].
May 17CHMP requests supplementary data from Santhera and the company now expect an opinion from the Committee during Q3/2017 [13].
Jun 16Filed in EU for treating patients with DMD who have respiratory function decline and who are not taking concomitant glucocorticoids [10].
Apr 15FDA has granted idebenone fast track status for DMD [9].
Mar 15Discussion with regulatory agencies continuing [7].
Apr 14Santhera Pharmaceuticals reported in September 2013 that it was continuing to explore strategic options and to seek additional financing [5].
Sep 09Orphan drug status in EU and US [2].
Sep 09Both the FDA and EMEA have agreed that, subject to positive outcome, a single pivotal study could suffice for approval [1].
Sep 09PIII study started [1].


Mitochondrial membrane transport protein stimulant
One of the most common and devastating types of muscular degeneration. Affects boys of all ethnicities from as early as 3-5 years of age with av life expectancy of 30-35 years. About 30,000 males in Europe and N. America have DMD [1]
Duchenne muscular dystrophy
to slow decline in respiratory function

Further information


Trial or other data

Sep 16PIII trial (NCT 02814019, SIDEROS) launched to investigate efficacy of idebenone in patients with Duchenne muscular dystrophy in delaying the loss of respiratory function in patients who are receiving concomitant glucocorticoids [11].
Oct 15Current options (corticosteroids) can delay but not prevent loss of walking ability. If licensed, idebenone would potentially be suitable for all affected patients with DMD. Likely to be expensive.
Apr 15The PIII DELOS study published in the Lancet. In this 1-year study of 64 patients (10-18 years) with Duchenne muscular dystrophy treatment with idebenone (300mg three times a day) attenuated the fall in peak expiratory flow (as percentage predicted) versus placebo (difference of -6.27%; [0.61 to 11.93]; p=0.031). The effect of idebenone on respiratory function outcomes was similar between patients with previous corticosteroid use and steroid-naive patients [8].
May 14Santhera Pharmaceuticals (SIX:SANN) announces today that its PIII DELOS study met the primary endpoint of delaying the loss of respiratory function compared to placebo; difference between Catena®/Raxone® and placebo in the change from baseline to week 52 in Peak Expiratory Flow (p=0.04) [6].
Apr 13The PIII DELOS study has passed a planned futility and safety analysis; the Data Safety Monitoring Board considers the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids [4].
May 11Following a 12-month PII DELPHI study, participants were able to enter the DELPHI-E, a 24-month open-label study, and 19 did so. They received weight-adjusted doses of Catena® at 450/900 mg/day. On average, patients did not receive Catena® for 21 months between DELPHI and DELPHI-E studies. During this period, PEF, FVC and maximum inspiratory mouth pressures (MIP) declined. The rate of decline was numerically lower for PEF and MIP when patients received Catena® treatment during the DELPHI-E study vs the period where they did not receive Catena®. There was no change in the rate of decline in FVC between the off-medication period and the DELPHI-E study period. Comparing data from DELPHI-E with natural history data for Duchenne Muscular Dystrophy, patients in the DELPHI-E study performed better in PEF for ages 16 and older and in FVC for ages 18 and older. Catena® possibly slows the progression of respiratory dysfunction in these age periods [3].
Sep 09The PIIa DELPHI trial (SNT-II-001; NCT00654784) in 21 boys aged 8-16 years has demonstrated positive effects on cardiac and respiratory parameters with no difference in safety and tolerability vs idebenone and placebo. The trial is expected to complete in Feb 2011 [2].
Sep 09Takeda have an exclusive right to commercialise idebenone in the EU and Switzerland under the brand name Sovrima [1].
Sep 09The 12-month PIII DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) study has started and will enroll up to 240 patients (ambulatory and non-ambulatory) aged 10 to 18 years in 25 centres in EU and US and will compare idebenone 900 mg/day vs placebo. The primary endpoint is the change from baseline to week 52 in respiratory function measured by peak expiratory flow. Secondary endpoints include other respiratory parameters, muscle strength and motor function over the treatment period and improvement in quality of life [1].

Evidence based evaluations