FDA OKs Ketamine For Treating Organ Transplant Injuries With 'Orphan Drug' Status

The U.S. Food and Drug Administration (FDA) has granted PharmaTher Holdings Ltd. PHRRF an Orphan Drug designation for ketamine in the prevention of injury from organ transplantation.

Orphan drug designation, which grants special status to a sponsor’s drug or biological product to treat a rare disease or condition (defined as those affecting fewer than 200,000 people in the U.S.), would qualify ketamine for certain benefits and incentives, including:

• Seven years of marketing exclusivity
• Potential tax credits for certain clinical drug testing costs
• Eligibility for orphan drug grants
• And the FDA’s New Drug Application waiver filing fee of about $2.4 million.

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This new FDA grant adds to PharmaTher’s three other orphan drug designations for ketamine therapy: one for the treatment of “status epilepticus,” a rare neurological disorder requiring emergency treatment for a seizure; another for the treatment of amyotrophic lateral sclerosis, a progressive neuromuscular disease with a life expectancy of two to six years after diagnosis with no known cure; and another for the treatment of complex regional pain syndrome, a debilitating condition characterized by severe burning or throbbing pain in a limb.

According to the U.S. organ procurement and transplantation network, 41,355 solid organ transplants were made in 2021 and 39,241 solid organ transplants in 2022 so far, with about 105,000 patients waiting in the country. 

The four most common organs transplanted are the liver, kidney, heart, and lung. An Ischemia-Reperfusion Injury (IRI) in organ transplantation can result in a higher incidence of acute and chronic rejection, long-term morbidity and mortality. 

Quickly restoring blood supply of ischemic organs is crucial for avoiding or reducing this injury, and strategies for reperfusion damage attenuation include ischemic pre- and postconditioning or machine perfusion, all which are costly, sometimes even technically challenging, and only partially effective at preventing or treating acute organ dysfunction.

Research (see 1, 2, 3, 4, 5) has so far shown ketamine lessens the injury from ischemia/reperfusion by inhibiting NF-κB, suppressing the production of proinflammatory cytokines as IL-6 and TNF-α, while also showing anti-inflammatory effects by inhibiting the reactivity of leukocytes.

Photo courtesy of Spotmatik Ltd on Shutterstock and Wikimedia Commons.