This is an important collaboration in the field of rare diseases

TRPharm continues its ambitious work focused on rare diseases without slowing down. TRPharm is pursuing ongoing collaboration with global pharmaceutical company R-Pharm to develop an original medicine for Familial Mediterranean Fever, one of the rare diseases seen in Turkey. TRPharm General Manager İsmet İnce, noting that they have begun clinical studies of the molecule they developed, emphasizes that they will complete the study by year-end and make it available to patients. Rare diseases, a global health concern, are defined as diseases occurring in 1 in 2,000 or less frequency, most of which are progressive, metabolic, chronic, and some potentially fatal. While rare diseases occur in 1 out of every 16 people in Turkey, approximately 5 million people in Turkey and roughly 350 million people worldwide are known to have a rare disease. Approximately 50% of patients are children. Healthcare sector leader TRPharm has been conducting work in the field of rare diseases since its establishment. [caption id="attachment_160183" align="aligncenter"] TRPharm General Manager İsmet İnce[/caption]

We are working to provide solutions for rare diseases in the MENA Region as well

TRPharm General Manager İsmet İnce, noting that rare diseases are more prevalent in Turkey due to consanguineous marriages, stated: "As TRPharm, we support medical awareness processes for rare diseases in Turkey. In this way, we help enable earlier diagnosis and faster, more successful initiation of treatment processes. Pediatric metabolic disorders, pediatric immunology, pediatric and adult rheumatology, cardiology, neurology and gastroenterology are among our focus areas. In addition to Turkey, we are working to provide solutions for rare diseases in the MENA Region. We received our first product authorization in the Gulf Region for our specialized rare disease product. In 2023, we also began clinical studies for the treatment of Fanconi Anemia," he explained.

One in every thousand people is a patient with Familial Mediterranean Fever

TRPharm is also conducting work with R-Pharm on an original medicine for the treatment of Familial Mediterranean Fever in Turkey. İnce, describing how they have begun clinical studies of the RPH104 (Goflikicept) molecule in collaboration with global pharmaceutical company R-Pharm, stated that phase 2 studies of the molecule, which is planned to be used especially in Colchicine-Resistant Familial Mediterranean Fever, are ongoing. Saying that they will complete the study by year-end and ensure that a molecule developed in Turkey reaches patients outside Turkey, İnce noted that since the molecule will be manufactured as a medicine in Turkey, it will be more accessible compared to imported medicines.

R-Pharm CMO Mikhail Samsonov made the following comments on the subject:

"Familial Mediterranean Fever is considered a rare disease. However, it is quite prevalent in some regions. While the frequency of occurrence in Turkey is around 1 case in 1,000, Colchicine-Resistant Familial Mediterranean Fever is around 5-10%. The efficacy of the Goflikicept molecule has already been demonstrated in other autoinflammatory diseases presented at major international conferences. A multinational clinical trial is currently ongoing for Familial Mediterranean Fever as well. We aim to ensure this innovative treatment reaches patients."