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Application for ODD for Crofelemer, a rare gastrointestinal pathology

By 23 September 2022October 21st, 2022No Comments

Napo Therapeutics, a pharmaceutical company whose goal is to develop therapies that make a difference in the quality of life of patients suffering from rare diseases and with a strong unmet medical need, for which there are currently no satisfactory tools for diagnosis, prevention or treatment, had already obtained for the Crofelemer the orphan drug designation for short bowel syndrome (SBS).

Milan, May 27, 2022 – Napo Therapeutics has submitted an application to the European Medicines Agency (EMA) for the recognition of Crofelemer’s orphan drug status for a rare form of chronic diarrhea (CDD) called microvillary inclusion disease (MVID). It is a very rare and serious intestinal genetic disease, which affects children from birth, condemning them to feed for life through parenteral therapy.
Orphan drugs are drugs intended for the treatment of such rare diseases as to make it difficult to implement a drug development and registration plan. For this reason, incentives have been defined by the European Union for the research, development and marketing of these molecules.

The submission to the EMA is the first phase of the process of recognizing this status. After validation of the submission, the evaluation process will begin which takes approximately 90 days from acceptance.

Napo Therapeutics is an independent pharmaceutical company, founded in 2021 with headquarters in Milan, whose goal is to develop therapies in Europe that make a difference in the quality of life of patients suffering from rare diseases and with a strong unmet medical need. there are currently no satisfactory tools for diagnosis, prevention or treatment.

“We are very pleased to have arrived at the presentation of the orphan drug designation application for Crofelemer for microvillary inclusion disease,” said Massimo Mineo, CEO of Napo Therapeutics. “If, as we hope, a positive opinion is given, Crofelemer will be able to benefit from regulatory incentives to start a development in order to respond to a significant need that has not yet found answers in this serious rare disease”.

“Congenital diarrheal disorders (CDD) are a group of chronic hereditary intestinal diseases with heterogeneous causes. Each type of CDD is therefore a different disease and with a different pathogenetic mechanism” underlined Martire Particco, Chief Medical Officer of Napo Therapeutics. “Microvillary inclusion disease (MVID) is part of this group of diseases. It is a rare genetic disease that is life-threatening for infants and children with this disease, with severe developmental delay and in the most severe cases. to death caused by severe secretory diarrhea. ”

Microvillary inclusion disease is characterized, like all other congenital diarrheal disorders, in addition to chronic diarrhea also by secondary symptoms, including massive dehydration, metabolic acidosis or alkalosis and malnutrition, symptoms that occur rapidly and can endanger life.

Napo Therapeutics

Napo Therapeutics is a “start-up” led by a team of highly experienced professionals: born from Napo Pharmaceuticals, a subsidiary of Jaguar Health, based in San Francisco, whose goal is the development, registration and marketing of products pharmaceuticals with active ingredients of plant origin, obtained respecting sustainability and designed for the global market, without exclusions, regardless of the social or economic status of a country.
Napo Therapeutics’ mission is to make Crofelemer, patented by Jaguar Health, available to patients in Europe for the treatment of rare diseases with a strong unmet medical need. A drug containing the active ingredient Crofelemer is already approved in the United States for different indications than those being studied and developed by Napo Therapeutics.
Crofelemer is an active ingredient of plant origin, as it is extracted and purified from the sap of the red bark, also known as “dragon’s blood”, of the Croton lechleri ​​tree that grows in the Amazon rainforest. Napo Pharmaceuticals has defined a sustainable collection program, according to fair trade practices, for Crofelemer to ensure a high degree of quality, sustainability and support for indigenous communities.
At the end of 2021, the company obtained from the EMA the designation of orphan drug for the molecule for the condition Short Bowel Syndrome (SBS).
Napo Therapeutics will be the licensee and exclusive distributor of Crofelemer in Central, Western and Eastern Europe (excluding Russia).

The unique values of Napo Therapeutics: the Napo way

The company is based on a circular action model: from the ecosystem it draws remedies for human health and in treating patients and is committed to preserving the ecosystem in full compliance with the principles of environmental and social sustainability. Napo Therapeutics wants to create a new relationship paradigm with all stakeholders, focusing on the needs and desires of the person: the human factor is the key on which the company bases its mission and intends to develop its business.
In this model, the following become fundamental elements:
  • responsibility towards the patient
  • respect for the patient’s dignity
  • sustainability of production