Scottish Medicines Consortium Approves Santhera's Raxone® (idebenone) for Restricted Use in Patients with LHON within NHS Scotland

Santhera Pharmaceuticals Holding AG / Scottish Medicines Consortium Approves Santhera's Raxone® (idebenone) for Restricted Use in Patients with LHON within NHS Scotland . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

Liestal, Switzerland, May 9, 2017 - Santhera Pharmaceuticals (SIX: SANN) announces approval by the Scottish Medicines Consortium for restricted use of Raxone^® (idebenone) in patients with LHON, with effect May 8, 2017.  

Patients with Leber's hereditary optic neuropathy (LHON), an extremely rare, inherited cause of blindness, could benefit from the only licensed treatment to date, following advice issued by the Scottish Medicines Consortium (SMC) under the ultra-orphan medicine process. The SMC accepted Raxone^® (idebenone) for restricted use by NHS Scotland in the treatment of visual impairment in adolescent and adult patients with LHON who are not yet blind (i.e., they do not meet the UK criteria to be registered as severely sight impaired). Raxone was accepted following consideration through SMC's Patient and Clinician Engagement (PACE) process, for medicines used at the end of life and for very rare conditions. Scotland is the first country in the UK to make Raxone available for the treatment of LHON. The SMC advice takes into account the benefits of a Patient Access Scheme (PAS), which improves the cost-effectiveness of Raxone.

LHON is a devastating disease that causes blindness mainly in young men during their late teens/early adulthood. Patients suddenly lose central vision and can no longer see fine details, read print or recognize faces. Within 12 months from onset, about 80% of untreated patients will have become legally blind. This is exceptionally debilitating and isolating in young adults, affecting their education, career plans and overall quality of life. Russell Wheeler, Trustee of the UK based LHON Society said: "Despite LHON being identified almost 150 years ago, the lack of any approved treatment for this devastating condition until now has led to many patients feeling helpless and abandoned by the health service. We applaud the pragmatism of the SMC's decision following a rigorous and transparent process. The availability of Raxone to affected families in Scotland is a welcome step towards avoiding preventable blindness in patients with LHON and we hope it will be followed by similar availability in other parts of the UK."