Tranilast for IPF recommended for orphan drug designation in EU
Status would help advance inhaled treatment candidate NXP002 in Europe

A committee of the European Medicines Agency (EMA) is recommending orphan drug status — a designation that aims to speed therapy development in rare diseases — for tranilast, the active ingredient in NXP002, an inhaled formulation from Nuformix for treating idiopathic pulmonary fibrosis (IPF).
A decision by the European Commission, which grants regulatory status in the European Union, is expected within 30 days.
NXP002 is a patented formulation that repurposes the antihistamine tranilast for direct delivery to the lungs — either as an add-on to standard treatments or on its own in patients who cannot take them — for use in IPF.
This positive opinion, from EMA’s Committee for Orphan Medicinal Products (COMP), was based on the medication’s potential benefit for patients, according to a company press release.
“We are delighted to receive news of the COMP’s positive opinion regarding NXP002’s eligibility for orphan drug designation in IPF, a high-mortality rare disease, in urgent need of new treatments,” said Dan Gooding, PhD, executive director of Nuformix.
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Orphan drug designation is granted by the European Commission to medications that target serious diseases affecting no more than 5 in 10,000 people in the EU. Such status comes with certain benefits, including exclusive marketing rights in the EU member countries.
Noting that the designation process “involves considerable scientific scrutiny,” Gooding said Nuformix believes “the opinion serves as powerful independent third-party validation of NXP002’s underlying scientific rationale and existing data supporting its potential efficacy in treating fibrotic lung diseases such as IPF.”
Fibrotic diseases are ones, like pulmonary fibrosis, in which scar tissue — medically known as fibrosis — thickens and hardens, often building up in the body. In IPF, a progressive lung disease, such scar tissue forms in the lungs, making it hard to breathe.
“In addition to this validation, there are numerous developmental and commercial incentives to securing [orphan drug designation] for NXP002, including 10 years marketing exclusivity, all of which would be transferable to future licensing partners,” Gooding said.
To be eligible for orphan drug status, a medication must be intended to treat, diagnose, or prevent a life-threatening or long-term debilitating rare disease, or be unlikely to generate a profit. It must also offer significant benefits over existing treatments, or be the only available treatment option.
The COMP said that NXP002 meets all the criteria for orphan drug designation and that there is enough evidence that NXP002 could bring significant benefit to IPF patients, per the release.
Tranilast is thought to work by blocking transforming growth factor beta (TGF-beta), a protein that promotes the production of extracellular matrix (ECM) proteins that contribute to lung scarring in IPF when present in excessive levels. The ECM is the network of proteins and other molecules that surrounds and supports cells in tissues.
The [EU committee’s] opinion serves as powerful independent third-party validation of NXP002’s underlying scientific rationale and existing data supporting its potential efficacy in treating fibrotic lung diseases such as IPF.
Preclinical studies showed that NXP002 could reduce the production of pro-fibrotic molecules in the lungs of rats.
While inconsistent results caused a delay in starting clinical studies, additional preclinical testing in a lab model of human lung tissue showed that using a high dose of NXP002 alongside standard anti-fibrotics almost completely eliminated the production of pro-fibrotic molecules. There were no signs that NXP002 could be toxic.
“We will now progress to applying for [U.S. Food and Drug Administration (FDA)] orphan drug designation. Given the EMA’s positive opinion, we are hopeful that the … FDA would conclude similarly,” Good said.