The FDA grants the orphan drug designation for Inflazome’s zomelide for the treatment of periodic syndromes associated with cryopyrin (CAPS) | Business

DUBLIN AND CAMBRIDGE, England and BRISBANE, Australia – (BUSINESS WIRE) – 14 Jul 2020 –

Inflazome (, the pioneering inflammasome biotechnology company that develops multiple drugs that stop harmful inflammation, today announces that it has been granted the orphan drug designation by the U.S. Food and Drug Administration (FDA) for Inzomelid in treatment of the periodic associated with the cryopyrin Syndrome (CAPS).

The designation of orphan drug 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 inflammasome NLRP3. Activation of the NLRP3 inflammasome is implicated in many diseases caused by chronic and harmful inflammation. Inflazome is trying to deal with this inflammation with one of its investigative drugs, Inzomelid, a potent and selective NLRP3 inflammasome inhibitor that can be taken orally.

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

Dr. Thomas Jung, Chief Medical Officer, Inflazome, commented,“Receiving the orphan drug designation from the FDA is a validation of one of our investigative drugs, Inzomelid, and of the pioneering work we have done with the NLRP3 inflammasome since our foundation. NLRP3 activation is implicated in a broad spectrum of diseases, of which CAPS is only one. Today’s news is a positive development for the CAPS patient community, for whom there are currently limited treatment options. We are looking forward to further developing Inzomelid and starting our Phase II CAPS study later this year. “

Inflazome is a biotechnology company that leads the development of orally available drugs to address the unmet clinical needs of inflammatory diseases by targeting inflammasomes. Inflammasomes are known to guide many chronic inflammatory conditions, from Parkinson’s and Alzheimer’s to asthma, inflammatory bowel disease, chronic kidney disease, cardiovascular disease, arthritis and NASH. Inflazome has two clinical trial drugs. Inzomelid is being developed as an orally available, brain penetrating drug and Somalix is ​​being developed as an orally available drug with peripheral limitation. Both will address unmet clinical needs in inflammatory conditions. Inflazome is headquartered in Dublin, Ireland, with offices in Cambridge, the United Kingdom and Brisbane, Australia.

Information on the NLPR3 Inflammasome

Activated NLRP3 acts as a “danger sensor” in the body to release the pro-inflammatory cytokines IL-1β, IL-18 and induce uncontrolled death of lytic cells (piroptosis). These processes lead to chronic inflammation and, as such, NLRP3 has been implicated in a large number of diseases.

Information on periodic syndromes associated with cryopyrin

Periodic syndromes associated with crypirin (CAPS), also called autoinflammatory syndromes associated with cryopyrin, are three diseases related to a defect NLRP3 gene. CAPS includes neonatal multisystem inflammatory disease (NOMID), Muckle-Wells syndrome (MWS) and familial cold autoinflammatory syndrome (FCAS). The differences between these diseases lie in the severity and organs involved.

Information on the designation of orphan drug

The Orphan Drug Designation Program provides orphan status to drugs and biological substances that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases / disorders affecting less than 200,000 people in the United States or that they affect more than 200,000 people but are not expected to recover the costs of developing and marketing a therapeutic drug.

Dr. Jeremy Skillington | VP Business Development

E: j.skillington@inflazome.comMedia:

United Kingdom and international | Ciara Martin / Tim Stamper

Ireland and international | Jonathan Neilan / Patrick Berkery




Copyright Business Wire 2020.

PUB: 14/07/2020 02:00 / DISCO: 14/07/2020 02:00

Copyright Business Wire 2020.


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