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Ema, Crofelemer obtains an additional orphan drug designation also for microvillary inclusion disease (MVID)

By 14 November 2022No Comments

After being designated for Short Bowel syndrome (SBS) last December 2021, Napo Therapeutics gains another major Crofelemer’s orphan drug status approval for the treatment of the very rare microvillary inclusion disease.
The condition, characterized by untreatable neonatal secretory diarrhea, affects children from birth, condemning them to feed for life through parenteral therapy and exposure to serious complications.

Milan, 13 october 2022 – The European Medicines Agency (EMA) has granted orphan drug status to Crofelemer, a molecule developed by the pharmaceutical company Napo Therapeutics, for the treatment of microvillary inclusion disease (MVID), a rare form of diarrhea chronic (CDD). The decision comes at the end of the evaluation process started on May 19, 2022, with the submission to the EMA of the application for designation of orphan drug status.
Orphan drugs are drugs intended for the treatment of such rare diseases as to make it difficult to implement a development and registration plan. For this reason, incentives have been defined by the European Union for the research, development and marketing of these molecules.

Massimo Mineo, CEO of Napo Therapeutics comments:

“The designation of Crofelemer as orphan drug for microvillary inclusion disease (MVID) is an important achievement for our company to achieve the goal of responding to the need of thousands of people across Europe who are waiting to have access to therapeutic pathways. not incapacitating to face the pathology and be able to live a full life. Crofelemer has thus obtained in less than a year two designations as an orphan drug, placing itself as an innovative drug in the field of rare diseases. The next goal is the beginning of the registration clinical trial , and make this drug available as soon as possible to patients awaiting treatment”

With the aim of developing and marketing in Europe therapies that make a difference in the quality of life of patients and for which there are currently no satisfactory tools for diagnosis, prevention or treatment, it was born in 2021 < strong> Napo Therapeutics , a European start-up based in Milan, is led by a team of professionals with long experience in the world of BiG Pharma and with in-depth knowledge of the field of rare diseases and pathologies with a strong unmet medical needs .
Napo Therapeutics was born from Napo Pharmaceuticals , a subsidiary of Jaguar Health , based in San Francisco, whose goal is the development of pharmaceutical products with active ingredients of plant origin , dedicated to the treatment of rare diseases and for which there is a strong unmet medical need. The drugs are designed for a global market , with no exclusions, regardless of the social or economic status of a country and respecting the criteria of environmental and social sustainability .

Highlight Martire Particco , Chief Medical Officer of Napo Therapeutics:

“The results, in terms of regulatory recognition, that Crofelemer is achieving, an active ingredient obtained through the techniques of ethnobotany from a plant of the Peruvian Amazon, show, once again, how it is possible to take care of human health, also in the field of rare diseases, drawing the necessary tools from nature, to which we in turn return the protection and respect for the environmental and social ecosystem in which we operate”.


The active ingredient, which had already obtained orphan drug status at the end of 2021 by the EMA for the treatment of another rare condition – the condition Short Bowel Syndrome (SBS) – is an active ingredient of plant origin, 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 harvesting program for which, for each tree used at least another two or three are replanted </ strong>: since 2000 approximately 800,000 Croton lechleri ​​trees have been planted, in seven areas of Peru ; 40,000 between 2020 and 2021 alone.

In full compliance with the principles of sustainability, Napo Pharmaceuticals cooperates with the local populations of the Peruvian Amazon and is committed to fair trade practices </ strong >, in order to guarantee a high degree of quality and support for the indigenous communities that guard the ecosystem of the banks of the Napo River : territories where the cultivation of coca leaves destined for decades has been widespread to drug production.

The treatment of the MVID

Microvillary inclusion disease (MVID) is a rare form of chronic diarrhea (CDD) for which satisfactory therapeutic pathways do not yet exist in Europe in order to guarantee patients the right to live a full life. The chronically debilitating pathology affects children from birth, condemning them to feed for life through parenteral therapy . Today, in the European Union about 0.1 in 10,000 people suffer from MVID .