Deal brings first treatment for Friedreich's Ataxia

Although Friedreich's Ataxia (FRDA) is primarily a neurological disease, cardiac symptoms are well recognised to be a life-threatening complication of the disease. The most common cardiac manifestation is hypertrophic cardiomyopathy, which ultimately causes death in the majority of FRDA patients.

SNT-MC17/idebenone is a membrane associated antioxidant that according to recent literature also mediates electron transport in mitochondria of Friedreich's Ataxia patients which are deficient in certain enzymes of the electron transport chain.

There is no single molecular target (e.g. enzyme or receptor) for SNT-MC17/idebenone rather due to its physicochemical properties it acts at the inner mitochondrial membrane and protects cells from oxidative stress and improves energy production.

Its uptake into cells and its distribution intracellularly to the mitochondria is optimized to protect mitochondria from their shortage of certain electron transport enzymes as a consequence of frataxin mutations (which is the molecular cause of the disease)

A spokesman for Santhera​ told DrugResearcher.com​: "SNT-MC17/idebenone does not offer a cure for Friedreich's ataxia but we believe can address the cardiac symptoms which are a life-threatening complication of the disease and which ultimately cause death through hypertrophic cardiomyopathy in the majority of Friedrich's Ataxia patients."​

The clinical development for FRDA is about to enter a Phase III clinical study in Europe and will enter a Phase III study in the US shortly thereafter. The product has orphan drug designation in the US and Europe.

"Guidance meetings with the FDA and the EMEA have already taken place to discuss the study designs for our pivotal trials. However, the ultimate challenge is still to show that the drug is efficacious in well designed phase III trials which Santhera is about to initiate,"​ he commented.

Under the terms of the collaboration, Santhera will conduct all clinical development for regulatory approval in Europe and in the US. Takeda will support the development work and will obtain an exclusive license to market Idebenone (SNT-MC17) in the European Union and Switzerland.

In return Santhera will receive an upfront payment of €5 million, development milestones and royalties from Takeda.​ Financial terms were not disclosed. Santhera will supply drug product to Takeda for Europe.

Additionally, Santhera plans to market Idebenone (SNT-MC17) on its own in the US.

"The successful development and commercialisation of a medicine will, for the first time, allow for the treatment of Friedreich's Ataxia, a life-threatening neuromuscular disease for which currently no pharmaceutical therapies exist."​ said Yasuchika Hasegawa, president and chief operating officer of Takeda.

Traditionally, drug companies largely neglect rare diseases such as FRDA. With the cost of research and development as well as the anticipated numbers who would use the drug, there is little incentive to invest heavily in time and costs for relatively little reward.

At present there are no treatments approved for Friedrich's Ataxia, hence Santhera and Takeda's interest in bringing SNT-MC17/idebenone to the market. Currently the only help provided to Friedreich Ataxia patients is of supportive nature. Psychological support, prostheses, walking aids, wheelchairs, physical therapy, and speech therapy are important for maintaining an active lifestyle. Orthopaedic interventions for scoliosis and foot deformities frequently become necessary.

However, the spokesman said: "All patients, however rare their disease deserve help. In particular if researchers come up with an idea for the treatment of such a disease, we believe it should be followed up."​

"Both Santhera and Takeda feel that there is a high medical need for Friedreich Ataxia patients both to treat and extend the lives of these patients given the preclinical and clinical work that has been undertaken,"​ he added.