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PEGASEMP™, the lead product of TREAT U, received the Orphan Drug Designation for the treatment of malignant mesothelioma

09.05.2018 in Bluepharma

“PEGASEMP™ allows inhibition of tumor growth and prevents metastization of mesothelioma, with low incidence of side effects.



PEGASEMP™ is the lead product of TREAT U that has just received the Orphan Drug designation (ODD) in the treatment of malignant mesothelioma from the European Medicines Agency (EMA) and the American Regulatory Authority, Food and Drug Administration (FDA). The next stage of development is to test PEGASEMP™ in the treatment of patients with mesothelioma.

PEGASEMP™ is a nanoparticle with the ability to carry a drug and deliver it specifically inside the tumor cells, avoiding the healthy organs. This new mechanism of action allowed PEGASEMP™ to be more effective than the approved chemotherapy in inhibiting the growth of the tumor by 183-fold in the most common subtype of mesothelioma and by 2713-fold in the most aggressive subtype. These data, alongside with the evidence of decreased side effects were enough for the European and North American authorities to grant the ODD to PEGASEMP™ in the treatment of malignant mesothelioma.

Mesothelioma is a rare cancer, with 3,000 people diagnosed each year in the USA and less than 1 in 10,000 in Europe. In Portugal, between 2002 and 2014, there were 199 deaths due to mesothelioma (data from INE). This tumor is well known for its correlation with exposure to asbestos, a fiber now considered to be an early-stage carcinogen, used in household materials and in navy shipyards since 1950. Pleural mesothelioma is a cause-of-death for most of the workers exposed to asbestos in the past. This tumor, located around the lungs, has a prognosis of just 6 months and the peak of incidence of this disease is estimated for 2020-2025.

PEGASEMP™ can be in the market by this time, partly because the ODD will allow a faster clinical demonstration of its benefits in mesothelioma patients. Once it reaches the market, because it is an approved orphan drug, PEGASEMP™ will also benefit from 10 years of protection from market competition in Europe, and 7 years in the USA.

To Vera Moura, CEO of TREAT U,  “the Orphan designation is extremely important to our company, as it qualifies us for several incentives in clinical testing. This will be the next stage for TREAT U: first-in-human trials with PEGASEMP™ in patients with mesothelioma. This is a rare tumor with a very bad prognosis, related with exposure to asbestos. We expect PEGASEMP™ to change the life of these patients and make a huge impact on their overall survival.”