Clinuvel Pharmaceuticals (CLVLY)

[FarmaZutical]

This article clarifies it somewhat:
Sorry for the poor Google Translate quality. The original is in Italian.

Source:

http://www.osservatoriomalattierare.it/farmaci-orfani/11039-protoporfiria-eritropoietica-san-gallicano-di-roma-garantisce-la-distribuzione-del-farmaco

Translation:

Soon there may be important news. The current context, it is likely to trigger further discrimination among the sick. Errors that would sound like a sentence for hundreds of people who, without being able to rely on a drug therapy, would be forced to not being able to expose to direct sunlight; especially during the hottest months of the year.

Observatory for Rare Diseases has decided to shed light on the whole affair listening to the leaders of the Roman structure and a sufferer. Marta Branca, special commissioner of "San Gallicano", explains how it was possible to ensure the delivery of the drug: "We chose to act in order not to leave the patients alone, we are talking about people who were likely to have an impossible life. Obviously the decision to triple the price of the drug from the company can not be considered secondary; we still continue to raise awareness of the AIFA, the Ministry of Health and the Regions to bring more merciful the manufacturing company. I want to emphasize that we have made direct pressure on AIFA during these months. "

The ruling shows specifically how it was possible to unblock a stalled condition: "I am committed to ask the Latium region to be able to use the budget we had available for the current year, for many months we have blocked purchases of the product. We are therefore making efforts to bridge the difference between the old price demanded by the company and the new. Through the agreements you will be then possible to treat patients living in other regions. Currently there are doing against two residents in Tuscany. This means that by the end of the year, or directly the next year, the problem will reoccur. I for one am confident that the drug company can return to a position of dialogue with the institutions. Our structure is trying to do the maximum in spite of the recovery plan that burden on the health system of the Lazio ".

The doctor responsible for rare diseases at the "San Gallicano", Giovanni Leone, recognizes the importance of therapy for people suffering from erythropoietic protoporphyria and remembers how the Roman structure was essential to achieve the commercialization of the treatment: "In recent years we took part experimentation with conviction by providing an adequate number of patients to research needs. In our structure we have never had problems until last year; In fact, the drug was dispensed through the provisions included in the Law 648. Then the decision to triple the price from the company. Request that the manufacturers do not seem inclined to change in the short term. We want to commit to eliminate disparities and ensure continuity of care. "

But we need to clarify the exact number of Italian patients: "The interested public should be about 140 sick even if the number is underestimated: I think the real number is around 200 people. The cases may still increase. We are faced with a genetic disorder that occurs not always at birth or early in life. "

Gerardo Tiso is a patient-physician, a member of an association representing sufferers protoporphyria. Years ago he participated directly in the experiments carried out in Rome. Choice that allowed to market the drug worldwide based Afamelanotide, compound created in the laboratory that stimulates melatogenesi and therefore able to protect the skin from ultraviolet rays. John describes his experience: "I have a certain age and have lived when there was no kind of drug treatment. I had to wear huge hats or gloves in the warmer months by the year: from May to September everything became more difficult. The consequences were not trivial. Exposure to direct sunlight meant risking burns of first or second degree and having to stand at rest for two weeks. "

The Scenesse drug has revolutionized the lives of patients but you are likely to make a jump in the past: "We are receiving many reports as an association. Patients tell us how it is becoming increasingly difficult to obtain the drug. Now they can rest assured only residents in Lazio, Lombardy, Veneto and Tuscany. Residents in Lombardy can be followed by the general hospital Ca 'Granda at coordinated by Professor Sunday Cappellini center. The Venetian may instead be borne by Professor Mauro Alaibac of Padua University Hospital. Residents in other regions should contact San Gallicano. The structure is ready to meet our medical needs. We have the impression that they have become standard sick B. Until a few months ago the situation was much better. " According to the doctor the situation could change, however: "We were able to talk to the President of AIFA, Mario Melazzini. The Agency wants to get to a market that is useful to patients' needs. For this reason it is important to continue beating the path of dialogue with Clinuvel ".