The story behind a rare disease drug

The story behind a rare disease drug

No drug happens by chance: the contents of any medicine bottle are the fruit of years and years of research, challenging experiments, and painstakingly accurate clinical trials. The road to new treatments is paved with failures and errors before it eventually reaches a breakthrough capable of improving the lives of hundreds and thousands of people.

The story of our own rare orphan disease drug started back in 1950 when Professor Rita Levi Montalcini discovered the Nerve Growth Factor (NGF), a recombinant protein stimulating nerve cell growth and a cornerstone of our journey into rare diseases. For her thirty-year-long research into NGF’s mechanisms of action, Professor Montalcini, together with biochemist Stanley Cohen, was awarded a Nobel Prize for medicine in 1986, making her the first Italian woman to have accomplished such an achievement.

It wasn’t until decades later that the first therapeutic application of NGF saw the light, also thanks to Dompé pharmaceuticals. In 2010, Sergio Dompé, at the helm of the family business since 1985, decided to invest in the field of orphan diseases by acquiring the rights to develop, produce and market NGF-based treatments. This triggered studies, research, and a new line of tests on its industrial development. “Our active principle is based on a recombinant protein, which means that it is produced from genetically modified bacteria” explains Gaetano D’Anniballe, Biotech R&D Director at Dompé, who goes on to say: “More precisely, NGF is a growth factor, a structurally very complex protein with a high biological activity: However, as all growth factors, it is also very delicate. We are the first company ever to have created a process suitable for conversion to industrial-scale production”.

Success came in 2013 when we used one of the initial batches produced in GMP and therefore suitable for human use, to implement the first international experimentation of phase I/II on the use of recombinant human Nerve Growth Factor for the treatment of rare diseases. Having overcome the production challenge, we then had to tackle distribution. The drug produced in our plant in L’Aquila is sent to hubs to be shipped to the European, US, and Chinese markets. The transport of this product is anything but straightforward: the complete cure lasts several weeks, and patients must receive their doses within precise deadlines.

The entire cure may be compromised by a single day’s delay. Accurate management of each step in the process has enabled the prompt delivery of every single package, even during the Covid-19 emergency when the world came to a standstill. “It is common thinking that research projects have no practical outcome: nothing could be farther from the truth” underscores D’Annibale - “Here, in these laboratories, often far removed from real life, research is conducted to build a better future. Many attempts are unsuccessful, but these failures are the foundations on which achievements are built, those that improve the lives of thousands of people. And nothing is worth more than that”.

Dompé farmaceutici S.p.A. Single shareholder company / Share capital € 50.000.000,00
REA MI 289519 - A registered company in Milan, Italy / Tax code and VAT number IT00791570153