Santhera Submits Marketing Authorization Application to the EuropeanMedicines Agency for Puldysa® (Idebenone) in Duchenne Muscular Dystrophy

Pratteln, Switzerland, – Santhera Pharmaceuticals (SIX: SANN) announces that it has
submitted a marketing authorization application (MAA) for Puldysa® (idebenone) for the treatment of
respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) to the European
Medicines Agency (EMA). Santhera is seeking conditional marketing authorization (CMA).
The indication for Puldysa sought under CMA is the treatment of respiratory dysfunction in patients with
DMD who are not using glucocorticoids. The MAA is supported by data from Santhera’s Phase II (DELPHI)
study, the long-term DELPHI-Extension study, the pivotal Phase III (DELOS) study [1-5] and the recently
completed SYROS study, a collection of long-term data from patients who completed the DELOS study
and continued to be treated with idebenone for up to six years [6].
“The new data included in this regulatory submission confirm clinically relevant patient benefits and
long-term therapeutic efficacy with idebenone in patients with DMD. Thereby, we have closed earlier
data gaps and respond to requirements from the regulatory authorities,” said Kristina Sjöblom Nygren,
MD, Chief Medical Officer and Head of Development at Santhera.
In line with previous communications, Santhera plans to submit a new drug application (NDA) with the
US FDA following the completion of the currently ongoing double-blind, placebo-controlled Phase III
(SIDEROS) study that investigates the efficacy and safety of idebenone in delaying the loss of respiratory
function in patients with DMD taking glucocorticoid steroids over a period of 18 months
( Identifier: NCT02814019; With 266 patients to be enrolled,
SIDEROS isthe largest actively recruiting clinical trial in DMD. To date, the study has 214 patients enrolled
at approximately 60 clinical trial sites in Europe, the US and Israel, with patient recruitment expected to
complete in 4Q 2019. Eligible patients who complete SIDEROS are offered to enroll in the open-label
SIDEROS-Extension study ( Identifier: NCT03603288).
Idebenone for the treatment of DMD has been granted orphan drug designation by European, US, Swiss
and Australian authorities, and it has fast track designation in the US.
[1] Buyse et al. (2015), The Lancet 385:1748-1757
[2] McDonald et al. (2016), Neuromuscular Disorders 26:473-480
[3] Buyse et al. (2017), Pediatric Pulmonology 52:580-515
[4] Mayer et al. (2017), Journal of Neuromuscular Diseases 4:189-198
[5] Buyse et al. (2018), Journal of Neuromuscular Diseases 5: 419-430
[6] Posters presented at the 2019 MDA Clinical & Scientific Conference
Santhera Submits Marketing Authorization Application to the European Medicines Agency
for Puldysa® (Idebenone) in Duchenne Muscular Dystrophy
May 27, 2019 / Page 2 of 3
About Duchenne Muscular Dystrophy
DMD is one of the most common and devastating types of progressive muscle weakness and
degeneration starting at an early age and leading to early morbidity and mortality due to respiratory
failure. It is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of
up to 1 in 3,500 live male births worldwide. DMD is characterized by a loss of the protein dystrophin,
leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy
production in muscle cells. With age, progressive respiratory muscle weakness affecting thoracic
accessory muscles and the diaphragm causes respiratory disease, impaired clearance of airway
secretions, recurrent pulmonary infections due to ineffective cough, and eventually respiratory failure.
There is currently no treatment approved for slowing loss of respiratory function in patients with DMD.
About Idebenone in Duchenne Muscular Dystrophy
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone
oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and
scavenging reactive oxygen species (ROS) and supplementing cellular energy levels.
DELOS was a Phase III, double-blind, placebo-controlled 52-week study which randomized 64 patients,
not taking concomitant steroids, to receive either idebenone (900 mg/day) or matching placebo. The
study met its primary endpoint, the change from baseline in peak expiratory flow (PEF) expressed as
percent of predicted, which demonstrated that idebenone can slow the loss of respiratory function.
Supportive data for idebenone were shown in the Phase II double-blind, placebo-controlled DELPHI study
and its 2-year open-label extension study (DELPHI-E).
SYROS was a prospectively planned, retrospective collection of long-term respiratory function data from
18 patients who completed the DELOS study and subsequently received idebenone (900 mg/day) under
Expanded Access Programs (EAPs). The SYROS study showed that the previously observed beneficial
effect of idebenone in reducing the rate of respiratory function decline was maintained for up to six years
during treatment.
About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the
development and commercialization of innovative medicines for rare and other diseases with high unmet
medical needs. The portfolio comprises clinical stage and marketed treatments for neuroophthalmologic, neuromuscular and pulmonary diseases. Santhera’s Raxone® (idebenone) is authorized
in the European Union, Norway, Iceland, Liechtenstein, Israel and Serbia for the treatment of Leber’s
hereditary optic neuropathy (LHON) and is currently commercialized in more than 20 countries. For
further information, please visit
Raxone® and Puldysa® are trademarks of Santhera Pharmaceuticals.
For further information please contact: or
Eva Kalias, Head External Communications
Phone: +41 79 875 27 80
Santhera Submits Marketing Authorization Application to the European Medicines Agency
for Puldysa® (Idebenone) in Duchenne Muscular Dystrophy
May 27, 2019 / Page 3 of 3
For Investors: or
Christoph Rentsch, Chief Financial Officer Hans Vitzthum, LifeSci Advisors
Europe: +41 61 906 89 65 US: +1 212 915 2568
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities
of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking
statements concerning the Company and its business. Such statements involve certain risks,
uncertainties and other factors which could cause the actual results, financial condition, performance or
achievements of the Company to be materially different from those expressed or implied by such
statements. Readers should therefore not place undue reliance on these statements, particularly not in
connection with any contract or investment decision. The Company disclaims any obligation to update
these forward-looking statements.
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Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on

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