FDA grants Orphan Drug Designation for Inflazome’s Inzomelid for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

July 14, 2020

Inflazomethe pioneering inflammasome biotech company developing multiple drugs that stop harmful inflammation, today announces that it has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for Inzomelid in the treatment of Cryopyrin-Associated Periodic Syndrome (CAPS). 


Orphan Drug Designation is an important regulatory milestone granted to drugs that are intended to treat rare diseases and conditions, such as CAPS. CAPS is a rare, autoinflammatory orphan disease driven by a mutation affecting the NLRP3 inflammasome. Activation of the NLRP3 inflammasome is implicated in many diseases caused by chronic, harmful inflammation. Inflazome is looking to address such inflammation with one of its investigative drugs, Inzomelid, a potent and selective inhibitor of the NLRP3 inflammasome that can be taken orally. 


In March 2020, Inflazome announced positive results with Inzomelid in CAPS, alongside the completion of a broader Phase I study that demonstrated excellent safety, tolerability and pharmacokinetics in healthy subjects. A follow-on Phase II trial is planned for this year to develop the dose for CAPS patients. 


Dr. Thomas Jung, Chief Medical Officer, Inflazome, commented, “Receiving Orphan Drug Designation from the FDA is a validation of one of our investigative drugs, Inzomelid, and the pioneering work that we have done with the NLRP3 inflammasome since our foundation. Activation of NLRP3 is implicated in a very wide spectrum of diseases, of which CAPS is just one. Today’s news is a positive development for the CAPS patient community, for whom there are currently limited treatment options. We look forward to further developing Inzomelid and initiating our Phase II study in CAPS later this year.” 


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