FDA Grants Orphan Drug Designation to Samumed’s Investigational SM04646 for IPF Treatment

FDA Grants Orphan Drug Designation to Samumed’s Investigational SM04646 for IPF Treatment

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SM04646, a therapy developed by San Diego-based Samumed to treat idiopathic pulmonary fibrosis (IPF). This designation provides several benefits, including tax incentives, exemption from the FDA user fee and potential market exclusivity for seven years following approval.

SM04646, given as a nebulized inhalation solution, was previously found to be well tolerated and safe in a Phase 1 clinical trial (ACTRN12615001349538).

Previously, in preclinical studies with an animal model of lung fibrosis, researchers found that aerosolized SM04646 reduced fibrosis-like alterations in lungs when compared to control treatment. Moreover, SM04646 showed more anti-fibrotic activity than the two already approved therapies for IPF — Esbriet (pirfenidone) and Ofev (nintedanib).

Samumed recently presented results of preclinical studies of SM04646, which demonstrated anti-fibrotic properties in numerous in vitro and in vivo studies, including a bleomycin-induced model of pulmonary fibrosis where aerosolized SM04646 reduced fibrosis in the lungs

SM04646 is believed to exert its anti-fibrotic action by decreasing the expression of genes related to fibrosis development. It has the potential for a dual application, either alone (monotherapy) or combined with currently approved oral medications, including Esbriet and Ofev.

“The FDA’s decision to grant an Orphan Drug Designation to SM04646 for IPF is another important milestone in the development of SM04646,” Yusuf Yazici, Samumed’s chief medical officer, said in a press release. “IPF is a chronic, progressive, fibrotic disorder that causes deteriorating lung function and severe dyspnea in patients and ultimately ends in fatality. Early trials demonstrate the therapeutic potential of SM04646 to help address the unmet medical need of individuals with IPF.”

In fact, IPF is the most common interstitial lung disease diagnosed by pulmonologists in the United States. The estimated median survival for IPF patients is between 2.5 and 3.5 years after diagnosis. The U.S. prevalence of IPF is 13.2 to 27.9 per 100,000 women, and 20.2 to 63.0 per 100,000 men.

Currently, IFP is incurable and available therapies are limited, necessitating the urgency for novel forms of therapy.






  1. Risa Sullivan says:

    Same question – when will it be available? Also, were there any side affects other than “no severe” ones? Thx!

  2. Cindi Buettner says:

    Would like information about any current or future trials for this drug . Also, base on prior new drugs, how long will it take this treatment to be released for prescription?

  3. Karen Charity says:

    We all want questions answered ASAP, Please.We have been waiting a long time for this. Some of us are worst off then others.Some have gone before us because there was no cure. So many need this desperately. Thank you

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