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.”
Inflazome is a biotech company leading the development of orally available drugs to address clinical unmet needs in inflammatory diseases by targeting inflammasomes. Inflammasomes are understood to drive 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-stage investigational drugs.
Inzomelid is under development as an orally available, brain-penetrant drug and Somalix is under development as an orally available, peripherally-restricted drug. Both will address clinical unmet needs in inflammatory conditions. Inflazome is headquartered in Dublin, Ireland, with offices in Cambridge, UK and Brisbane, Australia.
Activated NLRP3 acts as a ‘danger sensor’ in the body to release the pro-inflammatory cytokines IL-1β, IL-18 and induce uncontrolled, lytic cell death (pyroptosis). These processes lead to chronic inflammation, and as such, NLRP3 has been implicated in a large number of diseases.
Cryopyrin-associated periodic syndromes (CAPS), also called cryopyrin-associated autoinflammatory syndromes, are three diseases related to a defect in the NLRP3 gene. CAPS encompasses neonatal onset multisystem inflammatory disease (NOMID), Muckle-Wells syndrome (MWS) and familial cold autoinflammatory syndrome (FCAS). The differences in these diseases lie in their severity and the organs involved.
The Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Source: Company Press Release