Harmony Biosciences focuses on developing innovative treatments for neurological disorders, particularly those related to sleep and neurobehavioral conditions. Their patient-centric drug development model emphasizes the importance of patient insights in shaping clinical research programs. This approach is evident in their clinical trials, which include studies for conditions such as Prader-Willi Syndrome, Fragile X Syndrome, and Dravet Syndrome. Each trial is designed to rigorously evaluate the safety and efficacy of their products, ensuring that the needs of patients are at the forefront of their research efforts.

The company is also dedicated to raising awareness about sleep disorders, particularly narcolepsy, which often goes undiagnosed and carries significant stigma. Through initiatives like the "Global Wake-Up Call," Harmony Biosciences aims to educate the public and healthcare professionals about the challenges faced by individuals with narcolepsy. Their commitment extends beyond drug development to include community impact and philanthropy, reflecting a holistic approach to addressing the unmet medical needs of patients and their families.

United States


Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Rooted deep in empathy for patients’ needs and driven by novel science, visionary thinking, and an unwavering commitment to those who feel overlooked, Harmony Biosciences is helping patients thrive by cultivating the growth of innovation that’s essential to advancing life-changing treatments.

Visit us: https://www.harmonybiosciences.com/
Follow us: @Harmonybio on X and @harmony_biosciences on Instagram

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Genetic Diseases

Musculoskeletal Diseases

Nervous System Diseases

Psychological Diseases

Harmony Biosciences

Cataplexy

Excessive daytime sleepiness

Genetic disorder

Narcolepsy

Prader-Willi syndrome

WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse &nbsp;agonist. The mechanism of action of WAKIX is unclear; however, its &nbsp;efficacy could be mediated through its activity at H₃ receptors, thereby &nbsp;increasing the synthesis and release of histamine, a wake promoting neurotransmitter.

WAKIX® (pitolisant) 

WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019.&nbsp;It was granted orphan drug designation for the treatment of narcolepsy in 2010. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

Fragile X syndrome

Rare genetic disease

Zygel is the first and only pharmaceutically-produced CBD, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is being developed for patients suffering from FXS, ASD in pediatric patients, 22q, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE) (<a href="https://zynerba.com/in-development/zygel/">Learn more HERE</a>).&nbsp;

Zygel (ZYN002 CBD Gel) 

WAKIX® (pitolisant)

EDS in Idiopathic Hypersomnia

WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019.&nbsp;It was granted orphan drug designation for the treatment of narcolepsy in 2010. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

22q11.2 deletion syndrome

Deletion Syndrome

Zygel is the first and only pharmaceutically-produced CBD, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is being developed for patients suffering from FXS, ASD in pediatric patients, 22q, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE) (<a href="https://zynerba.com/in-development/zygel/" rel="noopener" target="_blank">Learn more HERE</a>).

Zygel (ZYN002 CBD Gel)

Muscle degeneration

Muscle weakness

Myotonic Dystrophy Type 1

Dravet syndrome

Lead product, clemizole hydrochloride (EPX-100), is a potent, oral, centrally-acting serotonin (5HT2) agonist

EPX-100 [clemizole hydrochloride]

Sleep-wake disorder

TPM-1116, a highly potent and selective oral orexin-2 receptor agonist that will be evaluated for the treatment of narcolepsy and other sleep-wake disorders.

Harmony Biosciences

Company Details