Systemic sclerosis, in Italy there is no life-saving drug that costs only a few cents

The doctors of Italian hospitals have issued a warning about the absence of one in their structures life-saving drug for patients suffering from scleroderma renal crisis due to systemic sclerosis. The missing drug in hospitals is, to be precise,ACE inhibitor in injectable solution, a medicine that does not owe its low quantities to the cost, but to the fact that Italian and European pharmaceutical companies are no longer interested in producing it.

Systemic sclerosis, there is no ACE inhibitor drug

To open the window on shortage of the ACE inhibitor drug in Italy he was the director of Immunorheumatology of the Biomedical Campus of Rome, Roberto Giacomelliwho recalled how until a few years ago patients suffering from systemic sclerosis they were treated with ACE inhibitors such as Enalapril, but now what is missing are the injectable vials – not the tablets – necessary to treat the pathology. “No one in Europe – said Roberto Giacomelli to Fatto Quotidiano – has asked himself the problem that it is a lifesaver, nor has the fund for rare diseases been activated. Yet there are gene therapies for rare diseases that cost tens of thousands of euros per month.” Even though it is a drug from low costabout 30 euro centsthe doctor underlines how European pharmaceutical companies have stopped producing it, with all that this entails in terms of poor availability for the care of sick people. “I believe – added Giacomelli – that even if they are no longer in production in Italy, the State should still have a supply of them”, with his appeal which was shared by the Italian Systemic Sclerosis League.

The importance of ACE inhibitors in injectable solution

There systemic sclerosis it is a chronic and progressive autoimmune disease, also known by the name of scleroderma, and was recognized in 2017 as a rare disease. In 10 – 15 percent of patients it may result inacute renal failure with the onset of moderate to marked hypertension. To prevent hypertension from proving fatal, aggressive treatment is needed that can stabilize and improve kidney function by up to 55 to 70 percent. To do this, however, as supported by Professor Roberto Giacomelli, you need ACE inhibitor antihypertensive drugs which are usually used to treat high blood pressure in minimal dosage. In the presence of a scleroderma renal crisis, the dosage is instead increased by 10 – 15 times, and requires intravenous administration. ACE inhibitor antihypertensive drugs, the doctor recalled, if administered in the early phase of the disease, have been shown to be able to prevent the most serious clinical manifestations. “These drugs – specified Giacomelli – in use since the early 90s, have allowed us to do reduce the mortality rate of scleroderma renal crisis from 80 percent to less than 10 percent. Their unavailability, therefore, takes us back to the lack of therapeutic options that existed in the 1980s.”

The mobilization of the Rare Diseases Observatory

To try to remedy the lack of life-saving drugs, theOmar, Rare Diseases Observatory raised the alarm together with the Italian Systemic Sclerosis League. One has been launched on petition to get the message to the message as quickly as possible Ministry of Health and to theAifa, Italian Medicines Agency. The competent ministry, as reported by Il Fatto Quotidiano, responded that it is studying solutions for important drugs and creating a national stockpile.