Health Care Professionals


Santhera is passionate about medical science and about developing new medical treatments for patients with rare and other diseases. Our focus is on the development of therapies for rare neuromuscular and pulmonary conditions with high unmet medical needs.

Our pipeline

Puldysa (idebenone) is being investigated for the treatment of Duchenne muscular dystrophy (DMD). In November 2018, Santhera acquired the option to exclusively in-license the first-in-class dissociative steroid vamorolone in all indications and all countries worldwide except Japan and South Korea. DMD is vamorolone’s lead indication. In February 2018, Santhera in-licensed from Polyphor a clinical stage selective inhibitor (POL6014) of human neutrophil elastase with the potential to treat cystic fibrosis (CF) and other lung diseases. In addition, Santhera’s pipeline includes omigapil, an investigational drug with anti-apoptotic properties, a compound in development to address unmet medical needs for patients with congenital muscular dystrophy (CMD).

Raxone in LHON

In September 2015, we received European Marketing Authorization for Raxone in the treatment of patients with Leber's hereditary optic neuropathy (LHON). This makes Raxone (idebenone 150 mg) the first and only medicine approved for this condition. Indeed, it is the first medicine approved for any mitochondrial disorder: a major milestone in addressing the unmet medical need represented by this group of rare diseases.

▼The European Medicines Agency (EMA) has indicated that this medicine is subject to additional monitoring.

Access to Raxone

Access to Raxone varies by the country from which the order is being placed. Bottles containing 180 tablets of Raxone (idebenone 150 mg) are currently available as follows:

European Union Member States, Norway and Iceland
For availability in one of the countries above please contact

Bosnia and Herzegovina, Montenegro, Republic of Macedonia (F.Y.R.O.M.), Serbia
For availability in these countries please contact Clinigen Group:
Phone: +44 1932 824 123

United States of America
Raxone/idebenone is NOT approved for use in the U.S. and is NOT available for purchase.

Note that Santhera is running a study with idebenone in patients with Duchenne muscular dystrophy (DMD) currently taking steroids that you/your child may be eligible for (see For additional information regarding this ongoing trial, please email

Santhera provides access to the investigational medicine idebenone for eligible patients 8 years of age and older with DMD through a U.S. Expanded Access Program (EAP) referred to as BreatheDMD, consistent with Santhera Pharmaceutical USA's policy on offering compassionate use to investigational medicines. Details regarding this EAP, including eligibility requirements, can be obtained by visiting the website

For medical inquiries regarding idebenone, please call: +1 800 887 6915.

All other countries not included above
For availability in all other countries not included above please contact Masters Speciality Pharma:
Phone: +44 208 327 0900
Fax: +44 208 327 0901