New Hope for Amyloidosis Patients Facing Organ Transplants or Dealing with Side Effects

Amyloidosis patients are expected to see some significant breakthroughs in treatment over the next three years, reducing side effects and possibly the need for organ transplants, research by healthcare market analysis specialist GlobalData has found.

Amyloidosis occurs when a build-up of proteins forms an abnormally folded structure within the body's cells and tissues, which then forms an insoluble deposit in a particular organ. Although amyloidosis is a severe and life-threatening disease, the market currently lacks an approved therapy.

The current therapeutic approaches towards amyloidosis are non-specific, invasive, often toxic and sometimes ineffective. The chemotherapeutic agents prescribed for the treatment of AL amyloidosis have poor safety profiles and sometimes cause patients to suffer more from side effects than the actual symptoms of the disease.

Treatment with chemotherapeutic agents is also disadvantageous to patients of senile and Aβ2M amyloidosis, leaving symptomatic treatments related to the heart or the cessation of dialysis as the only available options. Similarly, the existing therapies for AA and AF amyloidosis are limited to the control of the underlying inflammatory disease or liver transplantation.

The current off-label drugs prescribed to treat AL amyloidosis, such as melphalan, cyclophosphamide, vincristine, dexamethasone, prednisolone and Thalomid (thalidomide) and doxorubicin, lack efficacy and safety. As a result, the median survival of a patient with AL amyloidosis without treatment is approximately one year from diagnosis. Half of all patients diagnosed with AA amyloidosis die within five years of diagnosis. Without major treatment intervention, survival after the onset of AF amyloidosis is five to 15 years.

However, while the development of therapeutics to treat amyloidosis has been slow in recent years, pharmaceutical companies are now beginning to wake up to the commercial value of such studies. Although amyloidosis is rare, the cost of the drugs involved in the treatment of the disease is high enough to capture a significant share of the market. GlobalData's analysis shows that the value of the global amyloidosis therapeutics market currently stands at US$1.43 billion and is expected to reach US$2.58 billion by 2019.

As a result, the late-stage pipeline for the amyloidosis therapeutics market is currently pstrong, with 16 molecules in various phases of the drug development process. Of these molecules, four are in Phase III, six are in Phase II, four are in Phase I and two are in the pre-clinical stage. Eight are being tested for AL amyloidosis, three for AA amyloidosis and three for AF amyloidosis. In addition, Vyndaqel was approved in November 2011 by the European Medicines Agency (EMA) for the treatment of AF amyloidosis and is expected to be launched in the European market in early 2012, which may provide the breakthrough patients have waited for.