An October 2021 report published in the Globe and Newswire carried an announcement by Fabio Chianelli, CEO of the biotech company PharmaTher Holdings. The FDA approved orphan drug status for the drug ketamine to treat complex regional pain syndrome (CRPS). Ketamine has also been FDA-approved for the treatment of ALS.
About CRPS
One of the many features of ketamine is that it has been developed to treat complex regional pain syndrome (CRPS), a rare chronic inflammatory and pain condition. CRPS often occurs after an injury to either a leg, arm, hand, or foot.
CRPS may occur as a result of a spinal cord or brain injury or after surgery. Currently, there is no approved medication to treat CRPS.
CRPS is considered to be one of the most painful disorders. A study by Lee et al (Psychiatry Investig) found that thoughts of suicide occur more frequently in CRPS patients often reaching as high as seventy-five percent. The short and long-term effects of such excessive pain can result in permanent disability.
Hope For ALS Patients
The second FDA Orphan drug designation for ketamine was announced by the company in August 2021 for the treatment of Lou Gehrig’s disease (amyotrophic lateral sclerosis).
Currently, there are three medications for ALS that are approved by the FDA. However, they are not effective against the progression of the disease nor able to prolong the patient’s life span.
Preclinical research conducted at the University of Kansas Medical Center found that ketamine is capable of slowing muscle degeneration and may increase life expectancy in ALS patients.
The FDA’s orphan drug designation grants certain benefits such as tax credits and exclusive marketing rights. Ketamine is now heading towards a Phase II clinical trial.
About Esketamine, an Oral Antidepressant
Also, in March of 2019, the FDA granted the psychedelic drug company, PharmaTher Holdings (PharmaTher), the Orphan Drug designation for the nasal spray, esketamine (Spravato) to be combined with Esketamine, an oral antidepressant.
The drugs treat adults who have been unsuccessful in relieving their depression (termed treatment-resistant depression). As it is a chemical variant of ketamine and carries risks of adverse reactions and misuse, the FDA has attached severe restrictions to Esketamine. The restrictions include tight monitoring, the administration only under strict supervision, and only at certified locations.
About Ketamine
Ketamine is a dissociative anesthetic used in surgery. It causes memory loss and pain relief. When administered in low doses it produces stimulant effects. Medium or high doses can produce near-death experiences.
More About the FDA Orphan Drug Designation (ODD)
The ODS is granted to a drug that has been developed to treat a rare condition defined as affecting less than 200,000 individuals. Incentives include marketing exclusivity and tax credits for, among other incentives, clinical testing and a waiver of the New Drug filing fee of about $2.4 million.
PharmaTher is also initiating a phase 2 FDA-approved study of ketamine for Parkinson’s disease. In addition, it is developing a microneedle patch designed to administer psychedelics.
