PharmaTher Granted FDA Orphan Drug Designation for Ketamine to Treat Status Epilepticus

Rhea-AI Summary

PharmaTher Holdings Ltd. announced that the FDA granted Orphan Drug Designation for its ketamine product to treat Status Epilepticus (SE), a severe neurological condition requiring emergency treatment. This marks the third orphan designation for ketamine, adding to its portfolio for treating amyotrophic lateral sclerosis and complex regional pain syndrome. SE affects approximately 120,000-180,000 individuals annually in the U.S., with a mortality rate of 17%. Orphan status grants benefits including seven years of marketing exclusivity and potential tax credits.

Positive

• FDA granted Orphan Drug Designation for ketamine to treat Status Epilepticus, expanding its therapeutic potential.
• Adds to existing orphan designations for amyotrophic lateral sclerosis and complex regional pain syndrome.
• Orphan status provides incentives such as seven years of marketing exclusivity and tax credits.

Negative

• None.

Third FDA orphan drug designation for ketamine granted to PharmaTher

TORONTO, Feb. 01, 2022 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a leader in specialty ketamine pharmaceuticals, is pleased to announce that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) for ketamine to treat Status Epilepticus (“SE”), a rare neurological disorder requiring emergency treatment for a seizure.

The ODD for ketamine to treat SE expands the Company’s current FDA ODD portfolio for ketamine to treat amyotrophic lateral sclerosis and complex regional pain syndrome and strengthens the Company’s strategy in developing novel uses and delivery methods (i.e. microneedle patch) for ketamine to treat rare disorders and life-threatening conditions.

SE is a life-threatening occurrence of a prolonged seizure or recurrent seizures without recovery of consciousness between seizures (Lowenstein 1999) lasting more than five minutes. Epidemiological studies found an annual incidence of SE ranging from 41/100,000-61/100,000 (DeLorenzo 1996). Based on these studies, there are approximately 120,000-180,000 episodes of convulsive SE each year in the U.S. SE affects individuals of all ages and complicates various neurological and systemic illnesses. If SE is not treated immediately, permanent neuronal damage may occur, contributing to high morbidity and mortality rates. The mortality associated with SE is estimated at 17% and may lead to morbidity, including cognitive defects and neurological injury. SE is initially treated with benzodiazepines, of which approximately 35-45% of patients are refractory to benzodiazepines.

Fabio Chianelli, CEO of PharmaTher, said, “Studies have shown the potential of ketamine to treat various mental health, neurological and pain disorders. We are focused on becoming the leader for providing novel ketamine solutions and our dedication in expanding ketamine’s therapeutic utility in rare disorders and life-threatening conditions including, but not limited to, Parkinson’s disease, amyotrophic lateral sclerosis, complex regional pain syndrome, and now status epilepticus puts us on the right path in making ketamine more available to improve quality of life and to save lives.”

The Orphan Drug Act grants special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”). The FDA grants orphan status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at any given time. Orphan drug designation would qualify ketamine for certain benefits and incentives, including seven years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain clinical drug testing costs, activities, eligibility for orphan drug grants, and the waiver of the FDA New Drug Application filing fee of approximately $2.4 million.

About PharmaTher Holdings Ltd.

​PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) is focused on the development and commercialization of specialty ketamine pharmaceuticals for mental health, neurological, and pain disorders. Learn more at PharmaTher.com.

For more information about PharmaTher, please contact:

Fabio Chianelli
Chief Executive Officer
PharmaTher Holdings Ltd.
Tel: 1-888-846-3171
Email: info@pharmather.com
Website: www.pharmather.com

Neither the Canadian Securities Exchange nor its Regulation Services Provider have reviewed or accept responsibility for the adequacy or accuracy of this release.

Cautionary Statement

This press release contains 'forward-looking information' within the meaning of applicable Canadian securities legislation. These statements relate to future events or future performance. The use of any of the words "could", "intend", "expect", "believe", "will", "projected", "estimated", "potential", "aim", “may” and similar expressions and statements relating to matters that are not historical facts are intended to identify forward-looking information and are based on PharmaTher Holdings Ltd. (the "Company") current belief or assumptions as to the outcome and timing of such future events. Forward-looking information is based on reasonable assumptions that have been made by the Company at the date of the information and is subject to known and unknown risks, uncertainties, and other factors that may cause actual results or events to differ materially from those anticipated in the forward-looking information. Given these risks, uncertainties and assumptions, you should not unduly rely on these forward-looking statements. The forward-looking information contained in this press release is made as of the date hereof, and Company is not obligated to update or revise any forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable securities laws. The foregoing statements expressly qualify any forward-looking information contained herein. Factors that could cause actual results to differ materially from those anticipated in these forward-looking statements are described under the caption "Risk Factors" in Company's management's discussion and analysis for the period of November 30, 2021 ("MD&A"), dated January 27, 2022, which is available on the Company's profile at www.sedar.com.

This news release does not constitute an offer to sell or the solicitation of an offer to buy, and shall not constitute an offer, solicitation or sale in any state, province, territory or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state, province, territory or jurisdiction.

FAQ

What is the significance of the FDA granting Orphan Drug Designation to PharmaTher for ketamine?

The designation allows PharmaTher to pursue the development of ketamine for treating Status Epilepticus, providing benefits such as marketing exclusivity and tax incentives.

How many people are affected by Status Epilepticus in the U.S.?

Approximately 120,000 to 180,000 episodes of convulsive Status Epilepticus occur annually in the U.S.

What are the benefits of Orphan Drug Designation for PharmaTher's ketamine?

The designation offers seven years of marketing exclusivity, potential tax credits for clinical testing costs, and waiver of the FDA New Drug Application filing fee.

What other conditions is ketamine designated to treat by PharmaTher?

In addition to Status Epilepticus, ketamine is designated for treating amyotrophic lateral sclerosis and complex regional pain syndrome.