Orphan Drug Status Given to a New and Upcoming IPF Treatment

Recently, the U.S. FDA has deemed a particular idiopathic pulmonary fibrosis (IPF) drug “orphan drug status” worthy. Last week, on July 25, 2017, it was reported that the FDA granted orphan drug status to SM04646, which is a treatment/therapy created by Samumed, a biotech company in California.
I’m guessing that researchers there are pretty stoked about this designation because orphan drug status is basically the FDA granting special status to a drug or therapy in the treatment of rare diseases and conditions along with a bunch of added benefits, such as potential market exclusivity.
So what’s so great about this drug that warrants orphan drug status?

In a previous phase I clinical trial, SM04646 was deemed both safe and well tolerated for treatment of IPF, a rare disease in which the lungs harden from an unknown cause. Given as a nebulized inhalation solution, this therapy passed through all the necessary phase I checkpoints for IPF.

At the moment, IPF is the most common interstitial lung disease diagnosed by pulmonologists in the nation.

It is a progressive condition that results from the build-up of scar tissue, called fibrosis. This fibrosis eventually causes the lungs to lose their normal properties, like elasticity, so patients with IPF are less able to take oxygen in from surrounding air.

After diagnosis, there is an estimated mean survival for IPF patients between 2 and 3.5 years. Moreover, IPF does not have a cure and there are not many treatments available.

However, preclinical studies in animal models of lung fibrosis showed that SM04646 showed significant reduction in fibrosis-like changes in the lungs in comparison to the control treatment.
Even more importantly, perhaps, the treatment SM04646 demonstrated more anti-fibrotic activity than the other two approved IPF therapies (Esbriet and Ofev).
Scientists believe that SM04646 works its anti-fibrotic magic by reducing the expression of genes related to fibrosis development. In addition, this treatment has the ability to work either alone as a monotherapy, or combined with already approved oral treatment options (such as Esbriet and Ofev).
Chief Medical Officer of Samumed, Yusuf Yazici, is hopeful about this therapy in the treatment of IPF and feels that it has great potential to meet the current needs of individuals affected by IPF.

Thus, the push from the FDA for Samumed to finalize this new IPF treatment is both sensible and long-awaited. To read more about this new designation, as well as the treatment itself, click this link from Pulmonary Fibrosis News.

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