FDA grants ENA-001 orphan drug designation for Apnea of Prematurity


Eagle Pharmaceuticals and Enalare Therapeutics have announced that their ENA-001 drug for the treatment of Apnea of Prematurity has been granted an Orphan Drug Designation by the US Food and Drug Administration.

The US Food and Drug Administration has granted an Orphan Drug Designation (ODD) to Eagle Pharmaceuticals’ and Enalare Therapeutics’ ENA-001, a chemical entity for the treatment of Apnea of Prematurity (AoP).

AoP leads to immaturity of the pulmonary system, leading to breathing issues such as cessation of breathing for over 20 seconds or cessation of breathing for less than 20 seconds accompanied by bradycardia or hypoxemia.

About 25% of preterm infants are affected by AoP, with nearly all neonates with a gestational age of under 29 weeks or with a weight of less than 1000 grams being afflicted.AoP increases risk of mortality and long-term impaired neurological development.

ENA-001 treats AoP by inhibiting Big Potassium ion channels in the neck, stimulating breathing through the body’s ventilation control system.

Methylxanthines and caffeine are currently the main forms of treatment for AoP. ENA-001 will be a more advanced treatment option for preterm infants, according to Herm Cukier, president and CEO of Enalare Therapeutics.

ODD is granted to drugs which treat diseases that affect less than 200,000 people in the United States. Eligibility for tax grants, waiver of filing fees, research and development tax credits, and potential for a 7-year marketing exclusivity period are benefits available with ODD.

Funding for the development of ENA-001 has been provided by BARDA to develop treatments for infectious diseases. The National Institute on Drug Abuse of the National Institutes of Health has also given its support to the drug’s development.

Five human studies have indicated efficacy and tolerance of ENA-001. Enalare Therapeutics hopes the drug will be able to improve the lives of people affected by community drug overdose, post-operative respiratory depression, apnea of prematurity, and other life-threatening conditions.


Eagle Pharmaceuticals and Enalare Therapeutics announce FDA orphan drug designation for ENA-001 for the treatment of apnea of prematurity, a new chemical entity being developed as an agnostic respiratory stimulant. BioSpace. October 3, 2022. Accessed October 12, 2022. https://www.biospace.com/article/releases/eagle-pharmaceuticals-and-enalare-therapeutics-announce-fda-orphan-drug-designation-for-ena-001-for-the-treatment-of-apnea-of-prematurity-a-new-chemical-entity-being-developed-as-an-agnostic-respiratory-stimulant/

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