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Innovative Therapies

 

A committed to innovation

Dompé is committed to research, development and commercialization of innovative therapeutic solutions for rare and orphan diseases. A commitment the company carries out through a research network and a series of collaborations of excellence.

Rare diseases

The treatment of rare diseases is one of the main challenges of pharmaceutical research, but it is also an ideal instrument for generating knowledge and developing novel therapeutic approaches for common diseases. In fact, physio-pathological mechanisms can be found to be underlying factors of rare diseases, just as they are in the case of more common clinical conditions.

A rare disease is regarded as such in Europe when it affects less than 0.05% of the population, or five cases out of 10,000 people, in other words, an incidence that in Europe, though, corresponds to a total of about 30 million people1, while in the United States the US Food and Drug Administration (FDA) classifies a disease as rare when it affects no more than 200,000 people2. According to data from the World Health Organization, there are over 7,000 rare diseases, 80% of which have a genetic origin and 75% of which occur in childhood. In Europe, it is estimated that 6-8% of the population suffers from a rare disease, for a total ranging between 24 and 36 million people, while in the US it is estimated that people with a rare disease are about 25 million3.

To treat rare diseases, it is often necessary to develop specific drugs, which are referred to as orphan. These are medicinal products that may prove useful in treating a rare disease, but that for that very reason do not have a large enough market to cover the costs for developing them.

Thanks to an approach that is based on open innovation, Dompé has long been active in the research of new therapeutic solutions for the treatment of rare diseases. In fact, the Company conducts Research & Development programmes for the development of new drugs having an original mechanism of action, as it has already done in the field of ophthalmology.

The Company also markets a treatment for pulmonary arterial hypertension and a series of study treatments are in their advanced stage to improve the results of transplants of pancreatic cells in patients suffering from diabetes and in the treatment of early forms of this disease.

Biotechnology: present and future therapies

In the scientific field, biotechnology represents the “new frontier of innovation”: a seldom explored territory in which to find new responses to health-related questions that are still unanswered.

By biotechnology we mean all the technological applications used in living organisms or parts of them to produce useful substances for humans. Starting with recombinant human insulin, which was the first biotech drug to reach millions of patients with diabetes, today there are many areas of medicine in which biotech drugs are used, such as rheumatology, oncology, rare diseases, oncological haematology, and infectious diseases.

In the pharmaceutical industry, biotechnology is by all standards a field of excellence. A recent OECD study showed that in the countries surveyed, about 85% of the costs of R&D in biotechnology involve the pharmaceutical sector. In Italy, 90% of investments in R&D are funded by pharmaceutical companies, which have a research intensity 16 times higher than other high-medium technology sectors4.
Biotechnology is today a well-established and constantly evolving business in our country, where 202 biotech products are already available for the treatment of patients, the result of research conducted by 28 companies.

At its biotech plant, the Company has specialised in the development of new processes and in the production of biotech plant uhas been used for the production of recombinant proteins to treat rare diseases) and through an international network of partnerships in the field.

At its biotech plant, the Company has specialised in the development of new processes and in the production of biotech products on an industrial scale. The hub, which operates in compliance with GMP rules, is authorised to manufacture products using recombinant DNA technology, i.e. by transferring human genetic material to a bacterium, a process by which a protein can be produced that is fully complementary to human proteins.

Advanced therapies

Rapid and ongoing advances in biotechnology have led to the creation of new medicinal products that are made of cells and that have great potential in the treatment of several diseases.
The term advanced therapies includes three types of products: products for gene therapy, products for somatic cell therapy and tissue engineering products.

The first type of products are based on the transfer of genes, have a preventive, therapeutic or diagnostic action, and are created using recombinant Dna technology. Somatic cell therapy involves the use of cells or tissues which, thanks to research, can change their function. Finally, tissue engineering is based on the use of modified cells or tissues to regenerate or repair human tissue.

“Made in Italy” has a position of excellence in the field of advanced therapies, as three out of a total six authorised in Europe are Italian.

Dompé is highly active in the field both through the research it conducts and through a network of partnerships (such as the agreement with MolMed) for the marketing of advanced therapies.

 

 

References:

  1. Rare Disease Day 2015
  2. Rare Diseases: Common Issues in Drug Development Guidance for Industry
  3. Rare diseases
  4. Rapporto Annuale sulle Biotecnologia in Italia, Farmindustria (2017)