Updated on 24 December 2014
Mr Martin Andrews, senior vice president, Global Rare Diseases, GSK
Only a few nations like US and Europe get to enjoy the benefits of orphan drugs. Why are rare diseases an un-met medical need in Asia?
Unfortunately, the unmet needs in the US and in EU still remain considerable as only a fraction of patients have access to effective treatments. For instance, it is estimated that close to 30 percent of patients affected by the most common 350 rare diseases will die before they reach one year old. All patients with rare diseases, regardless of their geographic location, should have access to safe and effective orphan drugs.
Please highlight some of the major achievements of GSK in the orphan drug space.
We have a well-established history of successfully researching and developing medicines for rare diseases. Between 1983 to March 2010, GSK had the greatest number of orphan drug designations granted for medicines subsequently approved in the US when ranked against other pharmaceutical manufacturers.
The diversity of GSK drug discovery has yielded many programs with potential for rare disease indications; today 40 such programs exist with some 18 candidates in pre-clinical to mid stage development.
As a result of our recent efforts to help boys with Duchenne Muscular Dystrophy, we gathered the largest clinical dataset related to this condition in over 300 boys. In view of the limited information available on the natural history of rare diseases, we do hope that our contribution will go some way to help the scientific community's understanding of DMD.
One of our approaches is to investigate the potential of our existing medicines (generally for common diseases) for rare indications. As a result of this approach, we started a Phase III study of mepolizumab in patients with a rare disease called Eosinophilic Granulomatosis with Polyangiitis. EGPA - or Churg-Strauss syndrome as it was previously known - is a rare disease that causes widespread inflammation in the walls of small blood vessels. It can affect multiple organs and can be life threatening in some patients.
In October 2010 we signed an agreement with Fondazione Telethon and the Hospital San Raffaele in Italy to help children with several ultra-rare disorders. Our plans aim to help address three particular rare genetic disorders that we have exclusively in-licensed under this agreement (ADA-SCID, WAS and MLD) using gene therapy carried out on stem cells taken from the patient's bone marrow.
Only this week the results of the GSK/Gilead/Eli Lilly sponsored study, called the AMBITION study were presented at the 2014 European Respiratory Society (ERS) congress. This was the first study to investigate first line combination therapy of Volibris (ambrisentan) and Adcirca (tadalafil) in Pulmonary Arterial Hypertension (PAH). The study showed that the length of time before patients experienced clinical failure was significantly prolonged for those receiving first-line combination compared to monotherapy. We hope that these results may have a significant impact on the lives of patients suffering from PAH and we are now progressing plans to seek regulatory approval for this combination indication.